Trial Outcomes & Findings for Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (NCT NCT04562766)

Last Updated: 2026-02-23

Results Overview

Durable platelet response per guidance in regions except European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for \>=two-thirds of at least 8 non-missing weekly scheduled platelet measurements during the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy, provided that at least 2 non-missing weekly scheduled platelet measurements were at or above 50,000/mcL during the last 6 weeks of the 24-week blinded treatment period.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE3

Target enrollment

232 participants

Primary outcome timeframe

Up to 24 weeks

Results posted on

2026-02-23

Participant Flow

The study was conducted at 92 centers in 25 countries for adult participants and 20 centers in 11 countries for adolescent participants. 393 adult and 51 adolescent participants were screened between 14 December 2020 to 30 September 2024, of which 191 adult and 21 adolescent participants were screen failures. Screen failures were mainly due to not meeting eligibility criteria.

202 adult and 30 adolescent participants were randomized in the study. The study consisted of 3 periods: double-blind (DB) period, open-label (OL) period and safety follow-up or long-term extension (LTE) period. Interim results have been reported up to the database lock (DBL) date of 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period).

Participant milestones

Participant milestones
Measure	DB Period: Adults: Placebo Adult participants received placebo matched to rilzabrutinib tablet twice daily (BID) orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000 per microliter (/mcL) or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID Adult participants received rilzabrutinib 400 milligrams (mg) tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	OL Period: Adults: Rilzabrutinib 400 mg BID Eligible adult participants (responders after 24 weeks of DB period and non-responders after 12 weeks of DB period) entered the OL period to receive rilzabrutinib 400 mg tablet BID orally for 28 weeks.	OL Period: Adolescent: Rilzabrutinib 400 mg BID Eligible adolescent participants (responders after 24 weeks of DB period and non-responders after 12 weeks of DB period) entered the OL period to receive rilzabrutinib 400 mg tablet BID orally for 28 weeks.	LTE Period: Adults: Rilzabrutinib 400 mg BID Adult participants who completed the OL period and demonstrated a platelet response defined as: platelet counts \>=50,000/mcL or \>=30,000/mcL and at least doubled from baseline at \>=50% of the visits without receiving rescue therapy while on treatment during the last 8 weeks of the OL period were allowed to enter the LTE period and received rilzabrutinib 400 mg tablet BID orally in OL period. The maximum duration of the LTE period will be 12 months from the date of the last adult participant to enter the LTE.	LTE Period: Adolescent: Rilzabrutinib 400 mg BID Adolescent participants who completed the OL period and demonstrated a platelet response defined as: platelet counts \>=50,000/mcL or \>=30,000/mcL and at least doubled from baseline at \>=50% of the visits without receiving rescue therapy while on treatment during the last 8 weeks of the OL period were allowed to enter the LTE period and received rilzabrutinib 400 mg tablet BID orally in OL period. The maximum duration of the LTE period will be 12 months from the date of the last adolescent participant to enter the LTE.
DB Period (Weeks 1 to 24) COMPLETED	10	62	2	7	0	0	0	0
DB Period (Weeks 1 to 24) NOT COMPLETED	59	71	10	11	0	0	0	0
OL Period (From Week 25 to Week 53) STARTED	0	0	0	0	180	30	0	0
OL Period (From Week 25 to Week 53) COMPLETED	0	0	0	0	115	10	0	0
OL Period (From Week 25 to Week 53) NOT COMPLETED	0	0	0	0	65	20	0	0
LTE(Weeks 53-183[Adult]&147[Adolescent]) STARTED	0	0	0	0	0	0	69	4
LTE(Weeks 53-183[Adult]&147[Adolescent]) COMPLETED	0	0	0	0	0	0	1	0
LTE(Weeks 53-183[Adult]&147[Adolescent]) NOT COMPLETED	0	0	0	0	0	0	68	4
DB Period (Weeks 1 to 24) STARTED	69	133	12	18	0	0	0	0
DB Period (Weeks 1 to 24) Completed 12-week DB Period	66	121	12	18	0	0	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	DB Period: Adults: Placebo Adult participants received placebo matched to rilzabrutinib tablet twice daily (BID) orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000 per microliter (/mcL) or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID Adult participants received rilzabrutinib 400 milligrams (mg) tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	OL Period: Adults: Rilzabrutinib 400 mg BID Eligible adult participants (responders after 24 weeks of DB period and non-responders after 12 weeks of DB period) entered the OL period to receive rilzabrutinib 400 mg tablet BID orally for 28 weeks.	OL Period: Adolescent: Rilzabrutinib 400 mg BID Eligible adolescent participants (responders after 24 weeks of DB period and non-responders after 12 weeks of DB period) entered the OL period to receive rilzabrutinib 400 mg tablet BID orally for 28 weeks.	LTE Period: Adults: Rilzabrutinib 400 mg BID Adult participants who completed the OL period and demonstrated a platelet response defined as: platelet counts \>=50,000/mcL or \>=30,000/mcL and at least doubled from baseline at \>=50% of the visits without receiving rescue therapy while on treatment during the last 8 weeks of the OL period were allowed to enter the LTE period and received rilzabrutinib 400 mg tablet BID orally in OL period. The maximum duration of the LTE period will be 12 months from the date of the last adult participant to enter the LTE.	LTE Period: Adolescent: Rilzabrutinib 400 mg BID Adolescent participants who completed the OL period and demonstrated a platelet response defined as: platelet counts \>=50,000/mcL or \>=30,000/mcL and at least doubled from baseline at \>=50% of the visits without receiving rescue therapy while on treatment during the last 8 weeks of the OL period were allowed to enter the LTE period and received rilzabrutinib 400 mg tablet BID orally in OL period. The maximum duration of the LTE period will be 12 months from the date of the last adolescent participant to enter the LTE.
DB Period (Weeks 1 to 24) Withdrawal by Subject	2	7	0	0	0	0	0	0
DB Period (Weeks 1 to 24) Adverse Event	0	8	0	0	0	0	0	0
DB Period (Weeks 1 to 24) Participant was non-compliant to study requirements	1	0	0	0	0	0	0	0
DB Period (Weeks 1 to 24) Lack of response	55	55	10	11	0	0	0	0
DB Period (Weeks 1 to 24) Sponsor discretion	1	0	0	0	0	0	0	0
DB Period (Weeks 1 to 24) Other	0	1	0	0	0	0	0	0
OL Period (From Week 25 to Week 53) Withdrawal by Subject	0	0	0	0	21	4	0	0
OL Period (From Week 25 to Week 53) Adverse Event	0	0	0	0	6	0	0	0
OL Period (From Week 25 to Week 53) Lost to Follow-up	0	0	0	0	1	0	0	0
OL Period (From Week 25 to Week 53) Lack of response	0	0	0	0	33	6	0	0
OL Period (From Week 25 to Week 53) Other	0	0	0	0	4	0	0	0
OL Period (From Week 25 to Week 53) Ongoing	0	0	0	0	0	10	0	0
LTE(Weeks 53-183[Adult]&147[Adolescent]) Withdrawal by Subject	0	0	0	0	0	0	2	1
LTE(Weeks 53-183[Adult]&147[Adolescent]) Pregnancy	0	0	0	0	0	0	1	0
LTE(Weeks 53-183[Adult]&147[Adolescent]) Lack of response	0	0	0	0	0	0	1	1
LTE(Weeks 53-183[Adult]&147[Adolescent]) Other	0	0	0	0	0	0	1	0
LTE(Weeks 53-183[Adult]&147[Adolescent]) Sponsor discretion	0	0	0	0	0	0	1	0
LTE(Weeks 53-183[Adult]&147[Adolescent]) Adverse Event	0	0	0	0	0	0	1	0
LTE(Weeks 53-183[Adult]&147[Adolescent]) Ongoing	0	0	0	0	0	0	61	2

Baseline Characteristics

Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	DB Period: Adults: Placebo n=69 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo n=12 Participants Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID n=18 Participants Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	Total n=232 Participants Total of all reporting groups
Age, Continuous	47.1 years STANDARD_DEVIATION 18.0 • n=58 Participants	46.8 years STANDARD_DEVIATION 17.1	14.2 years STANDARD_DEVIATION 1.9 • n=1 Participants	14.6 years STANDARD_DEVIATION 1.4 • n=5 Participants	42.7 years STANDARD_DEVIATION 19.6 • n=8 Participants
Sex: Female, Male Female	49 Participants n=58 Participants	78 Participants	7 Participants n=1 Participants	9 Participants n=5 Participants	143 Participants n=8 Participants
Sex: Female, Male Male	20 Participants n=58 Participants	55 Participants	5 Participants n=1 Participants	9 Participants n=5 Participants	89 Participants n=8 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	1 Participants n=58 Participants	3 Participants	0 Participants n=1 Participants	0 Participants n=5 Participants	4 Participants n=8 Participants
Race/Ethnicity, Customized Asian	24 Participants n=58 Participants	40 Participants	8 Participants n=1 Participants	8 Participants n=5 Participants	80 Participants n=8 Participants
Race/Ethnicity, Customized Black or African American	0 Participants n=58 Participants	1 Participants	0 Participants n=1 Participants	1 Participants n=5 Participants	2 Participants n=8 Participants
Race/Ethnicity, Customized White	40 Participants n=58 Participants	85 Participants	3 Participants n=1 Participants	6 Participants n=5 Participants	134 Participants n=8 Participants
Race/Ethnicity, Customized Unknown or Not Reported	2 Participants n=58 Participants	1 Participants	0 Participants n=1 Participants	3 Participants n=5 Participants	6 Participants n=8 Participants
Race/Ethnicity, Customized Other	2 Participants n=58 Participants	3 Participants	1 Participants n=1 Participants	0 Participants n=5 Participants	6 Participants n=8 Participants

PRIMARY outcome

Timeframe: Up to 24 weeks

Population: Adult and adolescent intent-to-treat (ITT) population included all randomized participants.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=69 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo n=12 Participants Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID n=18 Participants Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom	0.0 percentage of participants Interval 0.0 to 0.0	23.3 percentage of participants Interval 16.12 to 30.49	0.0 percentage of participants Interval 0.0 to 26.46	11.1 percentage of participants Interval 1.38 to 34.71

PRIMARY outcome

Timeframe: Up to 24 weeks

Population: Adult and adolescent ITT population included all randomized participants.

Durable platelet response per guidance in European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=69 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo n=12 Participants Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID n=18 Participants Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom	0.0 percentage of participants Interval 0.0 to 0.0	23.3 percentage of participants Interval 16.12 to 30.49	0.0 percentage of participants Interval 0.0 to 26.46	11.1 percentage of participants Interval 1.38 to 34.71

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: Adult and adolescent ITT population included all randomized participants.

Number of weeks with platelet response was assessed using clinically meaningful threshold of platelet count \>=50,000/mcL or between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=69 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo n=12 Participants Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID n=18 Participants Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Number of Weeks With Platelet Count >=50,000/mcL or Between >=30,000/mcL and <50,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy	1.0 weeks Standard Deviation 2.14	7.5 weeks Standard Deviation 8.74	0.8 weeks Standard Deviation 1.54	4.2 weeks Standard Deviation 7.73

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: Adult and adolescent ITT population included all randomized participants.

Number of weeks with platelet response was assessed using clinically meaningful threshold of platelet counts \>=30,000/mcL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=69 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo n=12 Participants Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID n=18 Participants Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Number of Weeks With Platelet Counts >=30,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy	1.0 weeks Standard Deviation 2.01	7.3 weeks Standard Deviation 8.70	0.6 weeks Standard Deviation 1.08	4.1 weeks Standard Deviation 7.76

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: Adult and adolescent ITT population included all randomized participants. Only those participants who achieved platelet response are reported.

Time to first platelet count of \>=50,000/mcL or between \>=30,000/mcL and \<50,000/mcL and doubled from baseline was calculated as: (date of first occurrence of platelet response - date of first study drug intake) + 1.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=23 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=86 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo n=3 Participants Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID n=10 Participants Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Time to First Platelet Count of >=50,000/mcL or Between >=30,000/mcL and <50,000/mcL and Doubled From Baseline	NA days NA indicates that median and upper and lower limits of 95% confidence interval (CI) were not estimable due to insufficient number of participants with events during the DB period.	36.0 days Interval 22.0 to 44.0	NA days Interval 62.0 to NA indicates that median and upper limit of 95% CI were not estimable due to insufficient number of participants with events during the DB period.	74.5 days Interval 9.0 to NA indicates that upper limit of 95% CI was not estimable due to insufficient number of participants with events during the DB period.

SECONDARY outcome

Timeframe: Up to 24 weeks

Population: Adult and adolescent ITT population included all randomized participants.

Resue therapy included intravenous immunoglobulin (Ig) or high-dose corticosteroids, platelet infusion, or anti-D Ig infusion. Percentage of participants who required rescue therapy are presented.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=69 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo n=12 Participants Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID n=18 Participants Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Percentage of Participants Who Required Rescue Therapy	58.0 percentage of participants	33.1 percentage of participants	41.7 percentage of participants	27.8 percentage of participants

SECONDARY outcome

Timeframe: Baseline (Day 1) and Week 13

Population: Adult ITT population included all randomized participants.

ITP-PAQ:38 items and 44 items completed by male and female respondents respectively.It included 10 scales:symptoms(6 items:1-6),fatigue/sleep(4 items:7-10),bother-physical health(3 items:11-13),activity(2 items:14-15),psychological health(5 items:16-20),fear(5 items:21-25),overall quality of life(QoL)(5 items:26-30),social activity(4 items:31-34),women's reproductive health(6 items:35-40),work(4 items:41-44).Responses were recorded on 4-point(1:"strongly disagree" to 4:"strongly agree"),5-point(1:"never/not at all" to 5:"all the time/extremely") or 7-point(1:"not at all" to 7:"extremely") Likert scales.Each item score:100x(\[possible maximal item score - item score\]/range).All item scores:transformed to 0 to 100 continuum and were weighted equally to derive scale scores.Total score:0(worst) to 100(best);higher scores:better QoL.Change of positive value:improvement.Change from baseline in physical fatigue is presented.Baseline:last available value before first dose of DB study drug.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=69 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Change From Baseline on Item 10 (Physical Fatigue) of the Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) in Adult Participants at Week 13	-0.13 score on a scale Standard Error 2.861	7.95 score on a scale Standard Error 2.132	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Week 25

Population: Adult ITT population included all randomized participants.

Per guidance in European Union and United Kingdom, ITP IBLS was a bleeding assessment system which comprised of 11 (10 for male) site-specific grades assessed at 9 anatomical sites by history (Hx) over previous period. In addition, 2 of these sites, skin and oral, were also assessed by physical examination (PE). These 11 grades included: skin (PE), skin (Hx), oral (PE), oral (Hx), epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage; scores ranged from 0 (none) to 2 (marked bleeding). For each participant, an IBLS score at each visit was calculated by taking average across 11 items (10 for male and postmenopausal female) at 9 sites (8 for male and postmenopausal female). Total score was calculated as mean value for all 11 grades ranging from 0 (best) to 22 (worst); higher scores indicated worse outcomes. Change of negative value indicates an improvement.Baseline:last available value before first dose of DB study drug.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=69 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) Assessment in Adult Participants at Week 25 Per Guidance in European Union and United Kingdom	0.047 score on a scale Standard Error 0.0226	-0.040 score on a scale Standard Error 0.0169	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Week 25

Population: Adolescent ITT population included all randomized participants. Only those participants with data collected at specified timepoints are reported.

Per guidance in European Union and United Kingdom, ITP IBLS was a bleeding assessment system which comprised of 11 (10 for male) site-specific grades assessed at 9 anatomical sites by Hx over previous period. In addition, 2 of these sites, skin and oral, were also assessed by PE. These 11 grades included: skin (PE), skin (Hx), oral (PE), oral (Hx), epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage; scores ranged from 0 (none) to 2 (marked bleeding). For each participant, an IBLS score at each visit was calculated by taking average across 11 items (10 for male) at 9 sites (8 for male). Total score was calculated as mean value for all 11 grades ranging from 0 (best) to 22 (worst); higher scores indicated worse outcomes. Change of negative value indicates an improvement. Baseline:last available value before first dose of DB study drug.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=2 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=7 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale Assessment in Adolescent Participants at Week 25 Per Guidance in European Union and United Kingdom	-0.09 score on a scale Standard Deviation 0.13	-0.04 score on a scale Standard Deviation 0.15	—	—

SECONDARY outcome

Timeframe: Up to 52 weeks

Population: Adult rilzabrutinib safety population included all randomized participants who took at least 1 dose of study drug (rilzabrutinib), regardless of the amount of study drug (rilzabrutinib) administrated. The participants were evaluated from the start of DB period up to the end of OL period.

Stability of response was defined as the percentage of participants who were able to achieve stable platelet response defined as no 2 scheduled visits, at least 4 weeks apart, with a platelet count \<50,000/mcL, without an intervening visit with a platelet count \>=50,000/mcL, within a period of 24 weeks following initial achievement of the platelet response (initial platelet response defined as platelet count \>=50,000/mcL within 12 weeks of initiation of treatment with rilzabrutinib during the study). This endpoint was assessed from start of DB period through OL period.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=65 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo n=198 Participants Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB-OL Period: Percentage of Participants With Stability of Response in Adult Participants	23.1 percentage of participants Interval 12.83 to 33.32	23.3 percentage of participants Interval 16.12 to 30.49	23.2 percentage of participants Interval 17.35 to 29.11	—

SECONDARY outcome

Timeframe: Up to 52 weeks

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days (adults) and up to 175 days (adolescent)

Population: Adult and adolescent safety population included all randomized participants who took at least 1 dose of study drug, regardless of the amount of study drug administrated.

An AE was any untoward medical occurrence in a participant or clinical investigation participant, administered a study drug and which did not necessarily had a causal relationship with the study drug. An SAE was any AE that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. TEAEs were defined as AEs that developed, worsened or became serious during the TE period. Bleeding TEAEs Grade \>=2 (criteria mentioned in statistical analysis plan) are also presented.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=69 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo n=12 Participants Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID n=18 Participants Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs) and Bleeding Treatment-Emergent Adverse Events >=Grade 2 Any TEAE	52 Participants	111 Participants	10 Participants	15 Participants
DB Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs) and Bleeding Treatment-Emergent Adverse Events >=Grade 2 Any TESAE	8 Participants	12 Participants	0 Participants	2 Participants
DB Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs) and Bleeding Treatment-Emergent Adverse Events >=Grade 2 Any Bleeding TEAEs >=Grade 2	8 Participants	5 Participants	0 Participants	2 Participants

SECONDARY outcome

Timeframe: Pre-dose at Weeks 1, 13 and 25 and 2 hours post-dose at Week 1 and 25 (adults); Pre-dose at Weeks 1, 13 and 25, 0.5, 2, 4 and 6 hours post-dose at Weeks 1 and 25 (adolescent)

Population: Adult and adolescent pharmacokinetic (PK) population included all randomized and exposed participants (safety population) with at least 1 post-baseline PK sample. Only those participants with data collected at specified timepoints are reported.

Blood samples were collected at specified timepoints for the analysis of plasma concentration of rilzabrutinib.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=131 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=18 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Plasma Concentrations of Rilzabrutinib Week 13: Pre-dose	27.05 nanogram per milliliter (ng/mL) Standard Deviation 71.26	5.56 nanogram per milliliter (ng/mL) Standard Deviation 6.88	—	—
DB Period: Plasma Concentrations of Rilzabrutinib Week 25: Pre-dose	26.31 nanogram per milliliter (ng/mL) Standard Deviation 59.48	8.57 nanogram per milliliter (ng/mL) Standard Deviation 20.47	—	—
DB Period: Plasma Concentrations of Rilzabrutinib Week 1: Pre-dose	3.15 nanogram per milliliter (ng/mL) Standard Deviation 24.06	0.00 nanogram per milliliter (ng/mL) Standard Deviation 0.00	—	—
DB Period: Plasma Concentrations of Rilzabrutinib Week 1: 0.5 hour post-dose	—	34.51 nanogram per milliliter (ng/mL) Standard Deviation 40.24	—	—
DB Period: Plasma Concentrations of Rilzabrutinib Week 1: 2 hours post-dose	178.95 nanogram per milliliter (ng/mL) Standard Deviation 163.47	101.45 nanogram per milliliter (ng/mL) Standard Deviation 74.07	—	—
DB Period: Plasma Concentrations of Rilzabrutinib Week 1: 4 hours post-dose	—	52.62 nanogram per milliliter (ng/mL) Standard Deviation 33.70	—	—
DB Period: Plasma Concentrations of Rilzabrutinib Week 1: 6 hours post-dose	—	21.99 nanogram per milliliter (ng/mL) Standard Deviation 22.49	—	—
DB Period: Plasma Concentrations of Rilzabrutinib Week 25: 0.5 hour post-dose	—	81.27 nanogram per milliliter (ng/mL) Standard Deviation 70.06	—	—
DB Period: Plasma Concentrations of Rilzabrutinib Week 25: 2 hours post-dose	254.10 nanogram per milliliter (ng/mL) Standard Deviation 237.11	163.21 nanogram per milliliter (ng/mL) Standard Deviation 155.18	—	—
DB Period: Plasma Concentrations of Rilzabrutinib Week 25: 4 hours post-dose	—	72.23 nanogram per milliliter (ng/mL) Standard Deviation 66.37	—	—
DB Period: Plasma Concentrations of Rilzabrutinib Week 25: 6 hours post-dose	—	21.54 nanogram per milliliter (ng/mL) Standard Deviation 11.81	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Week 25

Population: Adult ITT population included all randomized participants. Only those participants with data collected at specified timepoints are reported.

ITP-PAQ:38 items and 44 items completed by male and female respondents respectively.It included 10 scales:symptoms(6 items:1-6),fatigue/sleep(4 items:7-10),bother-physical health(3 items:11-13),activity(2 items:14-15),psychological health(5 items:16-20),fear(5 items:21-25),overall QoL(5 items:26-30),social activity(4 items:31-34),women's reproductive health(6 items:35-40),work(4 items:41-44).Responses were recorded on 4-point(1:"strongly disagree" to 4:"strongly agree"),5-point(1:"never/not at all" to 5:"all the time/extremely") or 7-point(1:"not at all" to 7:"extremely") Likert scales.Each item score:100x(\[possible maximal item score - item score\]/range).All item scores:transformed to 0 to 100 continuum and were weighted equally to derive scale scores.Total score:0(worst) to 100(best);higher scores:better QoL.Change of positive value:improvement.Change from baseline in symptoms,bother and activity domains is presented.Baseline:last available value before first dose of DB study drug.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=10 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=60 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Change From Baseline in the Symptoms, Bother and Activity Domains of the Immune Thrombocytopenia-Patient Assessment Questionnaire in Adult Participants Symptoms	2.08 score on a scale Standard Deviation 12.92	10.28 score on a scale Standard Deviation 17.28	—	—
DB Period: Change From Baseline in the Symptoms, Bother and Activity Domains of the Immune Thrombocytopenia-Patient Assessment Questionnaire in Adult Participants Bother-physical health	-4.72 score on a scale Standard Deviation 19.69	9.98 score on a scale Standard Deviation 20.14	—	—
DB Period: Change From Baseline in the Symptoms, Bother and Activity Domains of the Immune Thrombocytopenia-Patient Assessment Questionnaire in Adult Participants Activity	-1.25 score on a scale Standard Deviation 25.99	9.96 score on a scale Standard Deviation 19.94	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Week 25

Population: Adolescent ITT population included all randomized participants. Only those participants with data collected at specified timepoints are reported.

ITP-KIT was a disease-specific instrument and child self-report form designed to be completed by children \>=7 years. It comprised of total of 27 items among which 26 items were structured as Likert scales with 5 response options 1: "never", 2: "rarely", 3: "sometimes", 4: "often" and 5: "always". An additional "not applicable" option was available for items 18 to 26, which was scored as 1: the same as "never". Item 27 was a descriptive question answered "yes" or "no" which was not included in the calculation of the summary score. Instrument yielded a summary KIT score which was the summation of the items calculated as: 100 x (1- \[{sum of all valid responses - number of valid responses}/{number of valid responses\*4}\]). Scores were converted to a 0 to 100 with higher scores indicating better disease-specific QoL. Change from baseline of positive value indicated improvement. Baseline was defined as the last available value before first dose of DB study drug.

Outcome measures

Outcome measures
Measure	DB Period: Adults: Placebo n=2 Participants Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Rilzabrutinib 400 mg BID n=7 Participants Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
DB Period: Change From Baseline in Disease-Specific Quality Of Life as Measured by the Kids' Immune Thrombocytopenia Tools (ITP-KIT) Score in Adolescent Participants	9.6 score on a scale Standard Deviation 6.8	2.0 score on a scale Standard Deviation 11.9	—	—

Adverse Events

DB Period: Adults: Rilzabrutinib 400 mg BID

Serious events: 12 serious events

Other events: 90 other events

Deaths: 1 deaths

DB Period: Adults: Placebo

Serious events: 8 serious events

Other events: 33 other events

Deaths: 0 deaths

DB Period: Adolescent: Placebo

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

DB Period: Adolescent: Rilzabrutinib 400 mg BID

Serious events: 2 serious events

Other events: 15 other events

Deaths: 0 deaths

OL Period: Adults: Rilzabrutinib 400 mg BID

Serious events: 17 serious events

Other events: 94 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 participants at risk Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Placebo n=69 participants at risk Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo n=12 participants at risk Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID n=18 participants at risk Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	OL Period: Adults: Rilzabrutinib 400 mg BID n=180 participants at risk Eligible adult participants (responders after 24 weeks of DB period and non-responders after 12 weeks of DB period) entered the OL period to receive rilzabrutinib 400 mg tablet BID orally for 28 weeks.
Infections and infestations Bronchopulmonary Aspergillosis	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Covid-19	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Cytomegalovirus Viraemia	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Pneumonia	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Renal Abscess	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Sepsis	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Urinary Tract Infection	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Wound Infection	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Blood and lymphatic system disorders Immune Thrombocytopenia	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Blood and lymphatic system disorders Thrombocytopenia	0.75% 1/133 • Number of events 4 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	2.9% 2/69 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	2.8% 5/180 • Number of events 12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Nervous system disorders Paraesthesia	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Eye disorders Conjunctival Haemorrhage	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Vascular disorders Deep Vein Thrombosis	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Vascular disorders Haematoma	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Vascular disorders Peripheral Embolism	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Respiratory, thoracic and mediastinal disorders Epistaxis	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Respiratory, thoracic and mediastinal disorders Interstitial Lung Disease	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Respiratory, thoracic and mediastinal disorders Pulmonary Embolism	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Haematemesis	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Mouth Haemorrhage	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Oesophageal Varices Haemorrhage	0.75% 1/133 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Hepatobiliary disorders Hepatic Cirrhosis	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Skin and subcutaneous tissue disorders Purpura	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Renal and urinary disorders Haematuria	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Reproductive system and breast disorders Heavy Menstrual Bleeding	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Reproductive system and breast disorders Ovarian Cyst Ruptured	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Reproductive system and breast disorders Vaginal Haemorrhage	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Investigations Hepatic Enzyme Increased	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Investigations Platelet Count Decreased	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Injury, poisoning and procedural complications Periorbital Haematoma	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Injury, poisoning and procedural complications Seroma	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Injury, poisoning and procedural complications Subdural Haematoma	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1

Other adverse events

Other adverse events
Measure	DB Period: Adults: Rilzabrutinib 400 mg BID n=133 participants at risk Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adults: Placebo n=69 participants at risk Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Placebo n=12 participants at risk Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	DB Period: Adolescent: Rilzabrutinib 400 mg BID n=18 participants at risk Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of \>=50,000/mcL or a platelet count of between \>=30,000/mcL and \<50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.	OL Period: Adults: Rilzabrutinib 400 mg BID n=180 participants at risk Eligible adult participants (responders after 24 weeks of DB period and non-responders after 12 weeks of DB period) entered the OL period to receive rilzabrutinib 400 mg tablet BID orally for 28 weeks.
Infections and infestations Covid-19	12.8% 17/133 • Number of events 17 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	4.3% 3/69 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	6.7% 12/180 • Number of events 13 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Hordeolum	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Influenza	3.0% 4/133 • Number of events 4 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.7% 3/180 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Lower Respiratory Tract Infection	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Nasopharyngitis	6.8% 9/133 • Number of events 9 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	2.9% 2/69 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	11.1% 2/18 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	10.0% 18/180 • Number of events 18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Pharyngitis Streptococcal	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Pneumonia	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Pneumonia Influenzal	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Sinusitis	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Infections and infestations Upper Respiratory Tract Infection	3.8% 5/133 • Number of events 10 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	4.3% 3/69 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	16.7% 2/12 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	11.1% 2/18 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	6.7% 12/180 • Number of events 14 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Blood and lymphatic system disorders Anaemia	3.8% 5/133 • Number of events 17 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.8% 4/69 • Number of events 4 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	4.4% 8/180 • Number of events 15 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Blood and lymphatic system disorders Blood Loss Anaemia	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Blood and lymphatic system disorders Coagulopathy	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Blood and lymphatic system disorders Iron Deficiency Anaemia	0.75% 1/133 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	4.3% 3/69 • Number of events 4 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	16.7% 2/12 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Blood and lymphatic system disorders Leukopenia	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Nervous system disorders Dizziness	8.3% 11/133 • Number of events 15 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	2.2% 4/180 • Number of events 5 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Nervous system disorders Headache	18.0% 24/133 • Number of events 52 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	7.2% 5/69 • Number of events 6 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	16.7% 2/12 • Number of events 5 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	11.1% 2/18 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 15/180 • Number of events 46 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Nervous system disorders Hypoaesthesia	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Nervous system disorders Presyncope	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.1% 2/180 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Ear and labyrinth disorders Ear Haemorrhage	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Vascular disorders Orthostatic Hypotension	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Respiratory, thoracic and mediastinal disorders Cough	4.5% 6/133 • Number of events 7 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	16.7% 2/12 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	11.1% 2/18 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.7% 3/180 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Respiratory, thoracic and mediastinal disorders Oropharyngeal Pain	2.3% 3/133 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	16.7% 2/12 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.1% 2/180 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Respiratory, thoracic and mediastinal disorders Rhinitis Allergic	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Respiratory, thoracic and mediastinal disorders Wheezing	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Abdominal Pain	7.5% 10/133 • Number of events 16 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	2.8% 5/180 • Number of events 6 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Abdominal Pain Upper	5.3% 7/133 • Number of events 10 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	11.1% 2/18 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	2.2% 4/180 • Number of events 4 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Constipation	1.5% 2/133 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.7% 3/180 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Diarrhoea	32.3% 43/133 • Number of events 72 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	10.1% 7/69 • Number of events 7 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	12.8% 23/180 • Number of events 33 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Dyspepsia	5.3% 7/133 • Number of events 7 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Nausea	20.3% 27/133 • Number of events 56 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.8% 4/69 • Number of events 4 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	33.3% 6/18 • Number of events 6 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	11.7% 21/180 • Number of events 27 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Stomatitis	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Toothache	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 4 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.7% 3/180 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Gastrointestinal disorders Vomiting	6.8% 9/133 • Number of events 13 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	16.7% 3/18 • Number of events 5 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	3.9% 7/180 • Number of events 7 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Skin and subcutaneous tissue disorders Rash Erythematous	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Musculoskeletal and connective tissue disorders Arthralgia	9.8% 13/133 • Number of events 16 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	4.3% 3/69 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	6.1% 11/180 • Number of events 13 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Musculoskeletal and connective tissue disorders Musculoskeletal Chest Pain	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Renal and urinary disorders Proteinuria	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.1% 2/180 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Reproductive system and breast disorders Dysmenorrhoea	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
General disorders Fatigue	3.0% 4/133 • Number of events 4 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.8% 4/69 • Number of events 4 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.7% 3/180 • Number of events 3 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
General disorders Oedema Peripheral	0.75% 1/133 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	2.2% 4/180 • Number of events 7 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
General disorders Pyrexia	3.8% 5/133 • Number of events 8 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	16.7% 2/12 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	2.8% 5/180 • Number of events 5 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Investigations Activated Partial Thromboplastin Time Prolonged	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Investigations Neutrophil Count Decreased	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/12 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	5.6% 1/18 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 2 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Investigations Urobilinogen Urine Increased	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/180 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Investigations Weight Increased	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.4% 1/69 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	1.7% 3/180 • Number of events 7 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Injury, poisoning and procedural complications Animal Bite	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Injury, poisoning and procedural complications Arthropod Bite	0.00% 0/133 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/69 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	8.3% 1/12 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.00% 0/18 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1	0.56% 1/180 • Number of events 1 • Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days. Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1

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