Trial Outcomes & Findings for A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy (NCT NCT04362761)
NCT ID: NCT04362761
Last Updated: 2023-07-28
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
172 participants
Primary outcome timeframe
From Day 1 up to Week 52
Results posted on
2023-07-28
Participant Flow
The study was conducted in 2 parts, Part A and Part B. Participants from Part A also participated in Part B. The Participant flow was planned to be presented for the overall treatment arm.
Participant milestones
| Measure |
ELX/TEZ/IVA
Part A: Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/tezacaftor (TEZ)100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) in the treatment period for 48 weeks.
Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks.
|
|---|---|
|
Part A (48 Weeks)
STARTED
|
172
|
|
Part A (48 Weeks)
COMPLETED
|
159
|
|
Part A (48 Weeks)
NOT COMPLETED
|
13
|
|
Part B (86 Weeks)
STARTED
|
50
|
|
Part B (86 Weeks)
COMPLETED
|
0
|
|
Part B (86 Weeks)
NOT COMPLETED
|
50
|
Reasons for withdrawal
| Measure |
ELX/TEZ/IVA
Part A: Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/tezacaftor (TEZ)100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) in the treatment period for 48 weeks.
Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks.
|
|---|---|
|
Part A (48 Weeks)
Adverse Event
|
2
|
|
Part A (48 Weeks)
Withdrawal of consent (not due to AE)
|
4
|
|
Part A (48 Weeks)
Commercial drug is available for participant
|
2
|
|
Part A (48 Weeks)
Other
|
5
|
|
Part B (86 Weeks)
Commercial drug is available for participant
|
46
|
|
Part B (86 Weeks)
Other
|
4
|
Baseline Characteristics
The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
Baseline characteristics by cohort
| Measure |
ELX/TEZ/IVA
n=172 Participants
Part A: Participants received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.
Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks.
|
|---|---|
|
Age, Continuous
Part A
|
27.9 years
STANDARD_DEVIATION 11.4 • n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Age, Continuous
Part B
|
25.6 years
STANDARD_DEVIATION 12.0 • n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Sex: Female, Male
Part A · Female
|
87 Participants
n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Sex: Female, Male
Part A · Male
|
85 Participants
n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Sex: Female, Male
Part B · Female
|
26 Participants
n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Sex: Female, Male
Part B · Male
|
24 Participants
n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · Hispanic or Latino
|
3 Participants
n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · Not Hispanic or Latino
|
167 Participants
n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · Not collected per local regulations
|
0 Participants
n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · Hispanic or Latino
|
1 Participants
n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · Not Hispanic or Latino
|
49 Participants
n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · Not collected per local regulations
|
0 Participants
n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · White
|
169 Participants
n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · Black or African American
|
0 Participants
n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · Asian
|
2 Participants
n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · American Indian or Alaska Native
|
0 Participants
n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · Native Hawaiian or Other Pacific Islander
|
0 Participants
n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · White, Asian
|
1 Participants
n=172 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · White
|
50 Participants
n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · Black or African American
|
0 Participants
n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · Asian
|
0 Participants
n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · American Indian or Alaska Native
|
0 Participants
n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · Native Hawaiian or Other Pacific Islander
|
0 Participants
n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · White, Asian
|
0 Participants
n=50 Participants • The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study.
|
PRIMARY outcome
Timeframe: From Day 1 up to Week 52Population: Safety set included all participants who received at least 1 dose of study drug.
Outcome measures
| Measure |
Part A: ELX/TEZ/IVA
n=172 Participants
Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.
|
|---|---|
|
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Participants with TEAEs
|
160 Participants
|
|
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Participants with SAEs
|
26 Participants
|
PRIMARY outcome
Timeframe: From Day 1 up to Week 86Population: Safety Set included all participants who received at least 1 dose of study drug.
Outcome measures
| Measure |
Part A: ELX/TEZ/IVA
n=50 Participants
Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.
|
|---|---|
|
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Participants with TEAEs
|
50 Participants
|
|
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Participants with SAEs
|
8 Participants
|
Adverse Events
Part A: ELX/TEZ/IVA
Serious events: 26 serious events
Other events: 127 other events
Deaths: 0 deaths
Part B: ELX/TEZ/IVA
Serious events: 8 serious events
Other events: 47 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Part A: ELX/TEZ/IVA
n=172 participants at risk
Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg qd in the treatment period for 48 weeks.
|
Part B: ELX/TEZ/IVA
n=50 participants at risk
Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg qd in the treatment period up to 86 weeks.
|
|---|---|---|
|
Cardiac disorders
Atrial fibrillation
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Cardiac disorders
Myocardial infarction
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Cardiac disorders
Sinus arrest
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Gastrointestinal disorders
Constipation
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Gastrointestinal disorders
Ileus
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
General disorders
Vascular device occlusion
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Hepatobiliary disorders
Cholecystitis
|
0.00%
0/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
2.0%
1/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Infections and infestations
Chronic sinusitis
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
4.7%
8/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
10.0%
5/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Infections and infestations
Lower respiratory tract infection
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Infections and infestations
Pneumonia
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Infections and infestations
Pneumonia pseudomonal
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Injury, poisoning and procedural complications
Limb traumatic amputation
|
0.00%
0/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
2.0%
1/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Injury, poisoning and procedural complications
Rib fracture
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Investigations
Alanine aminotransferase increased
|
1.2%
2/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Investigations
Aspartate aminotransferase increased
|
1.2%
2/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Investigations
Blood bilirubin increased
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Investigations
Forced expiratory volume decreased
|
0.00%
0/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
2.0%
1/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Investigations
Gamma-glutamyltransferase increased
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Metabolism and nutrition disorders
Electrolyte imbalance
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Musculoskeletal and connective tissue disorders
Dupuytren's contracture
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Nervous system disorders
Headache
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Nervous system disorders
Loss of consciousness
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Nervous system disorders
Seizure
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Psychiatric disorders
Aggression
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Psychiatric disorders
Dissociative disorder
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Reproductive system and breast disorders
Heavy menstrual bleeding
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Reproductive system and breast disorders
Uterine polyp
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal polyps
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
0.00%
0/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
2.0%
1/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Skin and subcutaneous tissue disorders
Erythema nodosum
|
0.58%
1/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
Other adverse events
| Measure |
Part A: ELX/TEZ/IVA
n=172 participants at risk
Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg qd in the treatment period for 48 weeks.
|
Part B: ELX/TEZ/IVA
n=50 participants at risk
Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg qd in the treatment period up to 86 weeks.
|
|---|---|---|
|
Investigations
Alanine aminotransferase increased
|
5.2%
9/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
2.0%
1/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Investigations
Aspartate aminotransferase increased
|
6.4%
11/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Investigations
Blood bilirubin increased
|
6.4%
11/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
2.0%
1/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Gastrointestinal disorders
Abdominal pain
|
9.3%
16/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
6.0%
3/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
7.0%
12/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
4.0%
2/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Gastrointestinal disorders
Constipation
|
5.8%
10/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
8.0%
4/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Gastrointestinal disorders
Diarrhoea
|
7.6%
13/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
4.0%
2/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
4.7%
8/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
6.0%
3/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Gastrointestinal disorders
Nausea
|
5.2%
9/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
2.0%
1/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Gastrointestinal disorders
Vomiting
|
4.7%
8/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
6.0%
3/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
General disorders
Fatigue
|
7.6%
13/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
2.0%
1/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
General disorders
Malaise
|
1.7%
3/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
6.0%
3/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
General disorders
Pyrexia
|
4.1%
7/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
16.0%
8/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Immune system disorders
Immunisation reaction
|
0.00%
0/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
14.0%
7/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Infections and infestations
COVID-19
|
1.2%
2/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
34.0%
17/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
9.9%
17/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
42.0%
21/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Infections and infestations
Influenza
|
1.2%
2/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
6.0%
3/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Infections and infestations
Nasopharyngitis
|
16.3%
28/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
32.0%
16/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Infections and infestations
Upper respiratory tract infection
|
5.8%
10/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
22.0%
11/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
1.2%
2/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
6.0%
3/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Injury, poisoning and procedural complications
Vaccination complication
|
14.0%
24/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.0%
12/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
8.0%
4/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Nervous system disorders
Dizziness
|
6.4%
11/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
0.00%
0/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Nervous system disorders
Headache
|
24.4%
42/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
20.0%
10/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
13.4%
23/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
10.0%
5/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
9.3%
16/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
10.0%
5/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
2.3%
4/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
12.0%
6/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
11.0%
19/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
10.0%
5/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.0%
12/172 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
4.0%
2/50 • Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 for Part B.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place