Trial Outcomes & Findings for Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B (NCT NCT02234310)
NCT ID: NCT02234310
Last Updated: 2022-03-25
Results Overview
Development of an inhibitor was defined as an inhibitor test result of \>= 0.60 Bethesda units per milliliter (BU/mL) that was confirmed by a second test result of \>=0.60 BU/mL from a separate sample, drawn 2 to 4 weeks after the date when the original sample was drawn, with both tests performed by the central laboratory using Nijmegen-modified Bethesda assay.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
33 participants
Primary outcome timeframe
Up to 3 years
Results posted on
2022-03-25
Participant Flow
The study was conducted at 28 active centers in 11 countries. Participants were screened between 13-Nov-2014 to 23-Jul-2018.
A total of 38 participants were screened. A total of 33 participants were enrolled, 22 participants began study participation on episodic treatment regimen and 11 participants began study participation on prophylactic treatment regimen.
Participant milestones
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
Participants received rFIXFc intravenous (IV) injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 International Units per kilogram (IU/kg) weekly until a participant reached at least 50 exposure days (ED=24-hour period in which greater than or equal to (\>=1) injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Overall Study
STARTED
|
33
|
|
Overall Study
Episodic Treatment Regimen
|
22
|
|
Overall Study
Prophylactic Treatment Regimen
|
28
|
|
Overall Study
COMPLETED
|
27
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
Participants received rFIXFc intravenous (IV) injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 International Units per kilogram (IU/kg) weekly until a participant reached at least 50 exposure days (ED=24-hour period in which greater than or equal to (\>=1) injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Eligibility Criteria not fulfilled
|
2
|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
Baseline characteristics by cohort
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=33 Participants
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Age, Continuous
|
0.60 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
33 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
White
|
22 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Black or African-American
|
1 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Unknown or Not reported
|
5 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Other
|
4 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Up to 3 yearsPopulation: Analysis performed on Safety Analysis set which included all participants who had received at least 1 dose of study treatment.
Development of an inhibitor was defined as an inhibitor test result of \>= 0.60 Bethesda units per milliliter (BU/mL) that was confirmed by a second test result of \>=0.60 BU/mL from a separate sample, drawn 2 to 4 weeks after the date when the original sample was drawn, with both tests performed by the central laboratory using Nijmegen-modified Bethesda assay.
Outcome measures
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=33 Participants
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
|
3.03 percentage of participants
Interval 0.08 to 15.76
|
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Analysis performed on Full Analysis Set (FAS) which included all enrolled participants with at least 1 dose of study treatment. Here, number analyzed signifies number of FAS participants who were analyzed in each treatment regimen.
ABR was annualized number of bleeding episodes during efficacy period (EP) per participant normalized to a 1-year interval of time. Bleeding episodes were classified as: spontaneous if parent/caregiver/participant records bleeding event when there is no known contributing factor such as definite trauma or antecedent strenuous activity; and traumatic when there is known reason for bleed. ABR=(Number of bleeding episodes during EP/total number of days during EP)\*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc per treatment regimens excluding surgical/rehabilitation periods and large injection intervals (greater than \[\>\]28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Outcome measures
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=33 Participants
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR])
Prophylaxis regimen
|
1.24 episodes per participant per year
Interval 0.0 to 5.4
|
|
Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR])
Episodic regimen
|
0.21 episodes per participant per year
Interval 0.0 to 6.8
|
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Analysis performed on FAS. Here, number analyzed signifies number of FAS participants who were analyzed in each treatment regimen.
Bleeding episodes were classified as spontaneous if parent/caregiver/participant records a bleeding event when there is no known contributing factor such as a definite trauma or antecedent "strenuous" activity. Annualized spontaneous joint bleeding episodes=(Total number of spontaneous joint bleeding episodes during efficacy period (EP) divided by total number of days during EP)\*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals (\>28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Outcome measures
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=33 Participants
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Annualized Number of Spontaneous Joint Bleeding Episodes
Episodic regimen
|
0.00 episodes per participant per year
Interval 0.0 to 5.6
|
|
Annualized Number of Spontaneous Joint Bleeding Episodes
Prophylaxis regimen
|
0.00 episodes per participant per year
Interval 0.0 to 2.6
|
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Analysis performed on FAS. Analysis was based on all injections. Here, number analyzed signifies number of responses to injections reported for each treatment regimen.
Using e-diary, each participant's parent/caregiver rated treatment response to any bleeding episode (BE) at approximately 8 to 12 hours from time of injection and prior to additional doses of rFIXFc given for same BE using 4-point scale:- 1=Excellent: abrupt pain relief and/or improvement in signs of bleeding within approximately 8 hours after initial injection; 2=Good: definite pain relief and/or improvement in signs of bleeding within approx. 8 hours after injection, but possibly requiring more than 1 injection after 24-48 hours for complete resolution; 3=Moderate: Probable/slight beneficial effect within 8 hours after initial injection and requires more than 1 injection and 4=None: No improvement or condition worsens within approx. 8 hours after initial injection. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Outcome measures
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=154 responses to injections
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
Episodic regimen · Excellent or Good
|
22 responses to injections
|
|
Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
Episodic regimen · Moderate
|
0 responses to injections
|
|
Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
Episodic regimen · None
|
0 responses to injections
|
|
Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
Episodic regimen · Response not provided
|
58 responses to injections
|
|
Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
Prophylaxis regimen · Moderate
|
6 responses to injections
|
|
Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
Prophylaxis regimen · None
|
1 responses to injections
|
|
Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
Prophylaxis regimen · Response not provided
|
17 responses to injections
|
|
Number of rFIXFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
Prophylaxis regimen · Excellent or Good
|
50 responses to injections
|
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Analysis performed on safety analysis set.
An ED was defined as a 24-hour period in which a participant received one or more doses of rFIXFc injections, with the time of the first injection of rFIXFc defined as the start of the ED. Participant who did not have a particular injection type were counted as having zero injections for that type.
Outcome measures
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=33 Participants
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Total Number of Exposure Days (EDs)
|
76.0 days
Interval 1.0 to 137.0
|
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Analysis performed on FAS. Here, number analyzed signifies number of FAS participants who were analyzed in each treatment regimen.
Total annualized rFIXFc consumption (in IU/kg) was calculated for each participant as: Annualized consumption = (Total IU/kg of rFIXFc during efficacy period (EP) divided by total number of days during EP)\*365.25. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (\> 28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Outcome measures
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=33 Participants
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Total Annualized rFIXFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes
Episodic regimen
|
203.2 IU per kilogram per participant per year
Interval 0.0 to 5719.0
|
|
Total Annualized rFIXFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes
Prophylaxis regimen
|
3175.0 IU per kilogram per participant per year
Interval 2544.0 to 13164.0
|
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Analysis performed on FAS. Here, number analyzed signifies number of bleeding episodes reported for each treatment regimen.
Number of injections of rFIXFc required to resolve a bleeding episode during efficacy period (EP) were reported. EP reflects the sum of all intervals of time during which participants were treated with rFIXFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (\>28 days). All injections given from the initial sign of a bleed, until the last date/time within the bleed window were counted. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Outcome measures
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=85 Bleeding episodes
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Number of Injections of rFIXFc Required to Resolve a Bleeding Episode
Episodic regimen
|
1.0 injections
Interval 1.0 to 31.0
|
|
Number of Injections of rFIXFc Required to Resolve a Bleeding Episode
Prophylaxis regimen
|
1.0 injections
Interval 1.0 to 4.0
|
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Analysis performed on FAS. Here, number analyzed signifies number of bleeding episodes reported for each treatment regimen.
The average dose of rFIXFc per injection per bleeding episode was calculated as the average of all doses (IU/kg) administered to treat the bleeding episode during efficacy period (EP). EP begins with the first treatment regimen dose of rFIXFc and ends with the last dose (regardless of the reason for dosing). Surgery/rehabilitation periods are not included in the EP. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Outcome measures
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=85 Bleeding episodes
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Average Dose Per Injection of rFIXFc Required to Resolve a Bleeding Episode
Episodic regimen
|
88.50 IU/kg
Interval 41.7 to 178.6
|
|
Average Dose Per Injection of rFIXFc Required to Resolve a Bleeding Episode
Prophylaxis regimen
|
71.92 IU/kg
Interval 23.3 to 181.8
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144Population: Analysis performed on FAS. Here number analyzed signifies number of FAS participants with available data for each visit.
Blood samples were taken at trough and Cmax (maximum concentration) for assessment of incremental recovery, measured by the one-stage clotting assay. IR (International Units per deciliter \[IU/dL\] per IU/kg) = (Cmax for FIX activity - Pre-dose FIX activity) (IU/dL)/ Actual dose (IU/kg), where Cmax is 30 minute FIX activity post-dose and FIX activity less than (\<)0.5 IU/dL was set to 0 IU/dL for calculation of IR.
Outcome measures
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=33 Participants
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 12
|
0.0 IU/dL per IU/kg
Interval -0.08 to 0.12
|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 24
|
0.0 IU/dL per IU/kg
Interval -0.17 to 0.15
|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 48
|
-0.0 IU/dL per IU/kg
Interval -0.13 to 0.03
|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 60
|
-0.1 IU/dL per IU/kg
Interval -0.1 to 0.03
|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 72
|
-0.1 IU/dL per IU/kg
Interval -0.2 to 0.07
|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 132
|
-0.1 IU/dL per IU/kg
Interval -0.1 to -0.1
|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 144
|
0.0 IU/dL per IU/kg
Interval 0.0 to 0.0
|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 36
|
-0.0 IU/dL per IU/kg
Interval -0.18 to 0.11
|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 84
|
-0.1 IU/dL per IU/kg
Interval -0.23 to -0.04
|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 96
|
-0.1 IU/dL per IU/kg
Interval -0.14 to 0.03
|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 108
|
-0.1 IU/dL per IU/kg
Interval -0.36 to -0.01
|
|
Change From Baseline in rFIXFc Incremental Recovery (IR)
Change at Week 120
|
-0.1 IU/dL per IU/kg
Interval -0.1 to -0.1
|
Adverse Events
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
Serious events: 23 serious events
Other events: 27 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=33 participants at risk
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Blood and lymphatic system disorders
Factor ix inhibition
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Infusion site pustule
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Blood and lymphatic system disorders
Immune thrombocytopenic purpura
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Congenital, familial and genetic disorders
Phimosis
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Gastrointestinal disorders
Inguinal hernia
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Gastrointestinal disorders
Tongue haemorrhage
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
General disorders
Vessel puncture site haematoma
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Immune system disorders
Hypersensitivity
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Croup infectious
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Gastroenteritis
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Lower respiratory tract infection
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Respiratory tract infection
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Staphylococcal bacteraemia
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Viral infection
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Viral rash
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Injury, poisoning and procedural complications
Accidental exposure to product
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Injury, poisoning and procedural complications
Craniocerebral injury
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Injury, poisoning and procedural complications
Face injury
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Injury, poisoning and procedural complications
Fall
|
15.2%
5/33 • Number of events 9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Injury, poisoning and procedural complications
Head injury
|
9.1%
3/33 • Number of events 8 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Injury, poisoning and procedural complications
Skull fracture
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Musculoskeletal and connective tissue disorders
Compartment syndrome
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Musculoskeletal and connective tissue disorders
Haemarthrosis
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Nervous system disorders
Coma
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Nervous system disorders
Febrile convulsion
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Nervous system disorders
Spinal cord haematoma
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Nervous system disorders
Tongue biting
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Reproductive system and breast disorders
Prepuce redundant
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Surgical and medical procedures
Central venous catheterisation
|
27.3%
9/33 • Number of events 9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Vascular disorders
Haematoma
|
3.0%
1/33 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Vascular disorders
Poor venous access
|
9.1%
3/33 • Number of events 3 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
Other adverse events
| Measure |
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
n=33 participants at risk
Participants received rFIXFc IV injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 IU/kg weekly until a participant reached at least 50 exposure days (ED=24-hour period in which \>=1 injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
|
|---|---|
|
Ear and labyrinth disorders
Middle ear effusion
|
6.1%
2/33 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Gastrointestinal disorders
Constipation
|
6.1%
2/33 • Number of events 3 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Gastrointestinal disorders
Diarrhoea
|
15.2%
5/33 • Number of events 9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Gastrointestinal disorders
Teething
|
18.2%
6/33 • Number of events 15 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Gastrointestinal disorders
Vomiting
|
12.1%
4/33 • Number of events 8 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
General disorders
Pyrexia
|
42.4%
14/33 • Number of events 33 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Bronchitis
|
6.1%
2/33 • Number of events 4 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Conjunctivitis
|
6.1%
2/33 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Croup infectious
|
6.1%
2/33 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Ear infection
|
12.1%
4/33 • Number of events 4 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Hand-foot-and-mouth disease
|
6.1%
2/33 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Influenza
|
9.1%
3/33 • Number of events 3 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Nasopharyngitis
|
33.3%
11/33 • Number of events 22 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Otitis media
|
12.1%
4/33 • Number of events 8 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Pharyngitis
|
12.1%
4/33 • Number of events 5 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Rhinitis
|
9.1%
3/33 • Number of events 5 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Upper respiratory tract infection
|
21.2%
7/33 • Number of events 12 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Varicella
|
12.1%
4/33 • Number of events 4 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Viral infection
|
12.1%
4/33 • Number of events 6 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Viral rash
|
9.1%
3/33 • Number of events 3 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
6.1%
2/33 • Number of events 3 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Injury, poisoning and procedural complications
Contusion
|
6.1%
2/33 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Injury, poisoning and procedural complications
Fall
|
21.2%
7/33 • Number of events 13 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Injury, poisoning and procedural complications
Head injury
|
15.2%
5/33 • Number of events 7 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Investigations
Blood alkaline phosphatase increased
|
9.1%
3/33 • Number of events 3 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Investigations
White blood cell count increased
|
6.1%
2/33 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Metabolism and nutrition disorders
Iron deficiency
|
9.1%
3/33 • Number of events 4 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Psychiatric disorders
Irritability
|
6.1%
2/33 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Reproductive system and breast disorders
Balanoposthitis
|
6.1%
2/33 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
6.1%
2/33 • Number of events 3 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
18.2%
6/33 • Number of events 11 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
6.1%
2/33 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
9.1%
3/33 • Number of events 3 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
18.2%
6/33 • Number of events 18 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
12.1%
4/33 • Number of events 5 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
6.1%
2/33 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
9.1%
3/33 • Number of events 4 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
6.1%
2/33 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
|
Skin and subcutaneous tissue disorders
Rash
|
9.1%
3/33 • Number of events 4 • All Adverse Events (AEs) were collected from signature of the informed consent form up to end of the study (up to 3 years) regardless of seriousness or relationship to investigational product.
Reported AE are treatment-emergent AE i.e. AE that was present prior to receiving first injection of rFIXFc and subsequently worsened in severity, or was not present prior to receiving first injection but subsequently appeared before last visit/follow-up call, whichever came later. Analysis performed on safety analysis set.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
- Publication restrictions are in place
Restriction type: OTHER