Trial Outcomes & Findings for Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years (NCT NCT04537793)
NCT ID: NCT04537793
Last Updated: 2023-06-28
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
83 participants
Primary outcome timeframe
From Day 1 through Day 15
Results posted on
2023-06-28
Participant Flow
The study was conducted in 2 parts, Part A and Part B. The Participant flow was planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen.
This study was conducted in participants with cystic fibrosis (CF) aged 2 through 5 years of age (inclusive).
Participant milestones
| Measure |
Part A: ELX/TEZ/IVA
Participants weighing greater than or equals to (\>=)14 kg at screening received elexacaftor (ELX) 100 milligrams (mg) once daily (qd)/tezacaftor (TEZ) 50 mg qd/ivacaftor (IVA) 75 mg every 12 hours (q12h) in the treatment period for 15 days.
|
Part B: ELX/TEZ/IVA
Participants weighing \>=14 kg at screening received ELX 100mg qd/TEZ 50mg qd/IVA 75 mg q12h and participants weighing \>=10 kg to less than (\<)14 kg received ELX 80mg qd/TEZ 40mg qd/IVA 60 mg once every morning (qAM) and IVA 59.5 mg once every evening (qPM) in the treatment period for 24 weeks.
|
|---|---|---|
|
Part A (15 Days)
STARTED
|
18
|
0
|
|
Part A (15 Days)
COMPLETED
|
18
|
0
|
|
Part A (15 Days)
NOT COMPLETED
|
0
|
0
|
|
Part B (24 Weeks)
STARTED
|
0
|
75
|
|
Part B (24 Weeks)
COMPLETED
|
0
|
74
|
|
Part B (24 Weeks)
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Part A: ELX/TEZ/IVA
Participants weighing greater than or equals to (\>=)14 kg at screening received elexacaftor (ELX) 100 milligrams (mg) once daily (qd)/tezacaftor (TEZ) 50 mg qd/ivacaftor (IVA) 75 mg every 12 hours (q12h) in the treatment period for 15 days.
|
Part B: ELX/TEZ/IVA
Participants weighing \>=14 kg at screening received ELX 100mg qd/TEZ 50mg qd/IVA 75 mg q12h and participants weighing \>=10 kg to less than (\<)14 kg received ELX 80mg qd/TEZ 40mg qd/IVA 60 mg once every morning (qAM) and IVA 59.5 mg once every evening (qPM) in the treatment period for 24 weeks.
|
|---|---|---|
|
Part B (24 Weeks)
Adverse Event
|
0
|
1
|
Baseline Characteristics
The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
Baseline characteristics by cohort
| Measure |
ELX/TEZ/IVA
n=83 Participants
Part A: Participants weighing greater than or equal to (\>=)14 kilograms (kg) at screening received ELX 100 milligrams (mg) once daily (qd)/TEZ 50 mg qd/IVA 75 mg every 12 hours (q12h) in the treatment period for 15 days.
Part B: Participants weighing \>=14 kg at screening received ELX 100mg qd/TEZ 50mg qd/IVA 75 mg q12h and participants weighing \>=10 kg to less than (\<)14 kg received ELX 80mg qd/TEZ 40mg qd/IVA 60 mg once every morning (qAM) and IVA 59.5 mg once every evening (qPM) in the treatment period for 24 weeks.
|
|---|---|
|
Age, Categorical
Part A · <=18 years
|
18 Participants
n=18 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Age, Categorical
Part A · Between 18 and 65 years
|
0 Participants
n=18 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Age, Categorical
Part A · >=65 years
|
0 Participants
n=18 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Age, Categorical
Part B · <=18 years
|
75 Participants
n=75 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Age, Categorical
Part B · Between 18 and 65 years
|
0 Participants
n=75 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Age, Categorical
Part B · >=65 years
|
0 Participants
n=75 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Sex: Female, Male
Part A · Female
|
11 Participants
n=18 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Sex: Female, Male
Part A · Male
|
7 Participants
n=18 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Sex: Female, Male
Part B · Female
|
41 Participants
n=75 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Sex: Female, Male
Part B · Male
|
34 Participants
n=75 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · Hispanic or Latino
|
0 Participants
n=18 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · Not Hispanic or Latino
|
18 Participants
n=18 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · Not collected per local regulations
|
0 Participants
n=18 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · Hispanic or Latino
|
6 Participants
n=75 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · Not Hispanic or Latino
|
63 Participants
n=75 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · Not collected per local regulations
|
6 Participants
n=75 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · White
|
18 Participants
n=18 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Race/Ethnicity, Customized
Part A · Black or African American
|
0 Participants
n=18 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · White
|
68 Participants
n=75 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
|
Race/Ethnicity, Customized
Part B · Black or African American
|
1 Participants
n=75 Participants • The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. Here "number analyzed" signifies participants who were evaluable for specified part of the study.
|
PRIMARY outcome
Timeframe: From Day 1 through Day 15Population: Pharmacokinetic (PK) set included participants who received at least 1 dose of study drug. Here the "Number Analyzed" signifies those participants who were evaluable at specified time point.
Outcome measures
| Measure |
Part A: ELX/TEZ/IVA
n=18 Participants
Participants weighing \>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days.
|
Part B: ELX/TEZ/IVA (≥14 kg)
Participants weighing \>=14 kg at screening received ELX100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 24 weeks.
|
|---|---|---|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 15: ELX-M23
|
2.47 micrograms per milliliter (mcg/ml)
Standard Deviation 1.65
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 1: ELX
|
0.00 micrograms per milliliter (mcg/ml)
Standard Deviation 0.00
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 8: ELX
|
3.44 micrograms per milliliter (mcg/ml)
Standard Deviation 2.09
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 15: ELX
|
3.69 micrograms per milliliter (mcg/ml)
Standard Deviation 2.11
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 1: ELX-M23
|
0.00 micrograms per milliliter (mcg/ml)
Standard Deviation 0.00
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 8: ELX-M23
|
2.39 micrograms per milliliter (mcg/ml)
Standard Deviation 1.65
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 1: TEZ
|
0.00 micrograms per milliliter (mcg/ml)
Standard Deviation 0.00
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 8: TEZ
|
1.86 micrograms per milliliter (mcg/ml)
Standard Deviation 1.07
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 15: TEZ
|
1.85 micrograms per milliliter (mcg/ml)
Standard Deviation 1.10
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 1: TEZ-M1
|
0.00 micrograms per milliliter (mcg/ml)
Standard Deviation 0.00
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 8: TEZ-M1
|
6.42 micrograms per milliliter (mcg/ml)
Standard Deviation 1.42
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 15: TEZ-M1
|
7.68 micrograms per milliliter (mcg/ml)
Standard Deviation 1.52
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 1: IVA
|
0.00 micrograms per milliliter (mcg/ml)
Standard Deviation 0.00
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 8: IVA
|
0.702 micrograms per milliliter (mcg/ml)
Standard Deviation 0.503
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 15: IVA
|
0.746 micrograms per milliliter (mcg/ml)
Standard Deviation 0.526
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 1: IVA-M1
|
0.00 micrograms per milliliter (mcg/ml)
Standard Deviation 0.00
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 8: IVA-M1
|
1.78 micrograms per milliliter (mcg/ml)
Standard Deviation 0.990
|
—
|
|
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Day 15: IVA-M1
|
1.91 micrograms per milliliter (mcg/ml)
Standard Deviation 0.990
|
—
|
PRIMARY outcome
Timeframe: From Day 1 up to Day 43Population: Safety set for Part A included all participants who received at least 1 dose of study drug in Part A. This outcome measure was planned only for Part A arm.
Outcome measures
| Measure |
Part A: ELX/TEZ/IVA
n=18 Participants
Participants weighing \>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days.
|
Part B: ELX/TEZ/IVA (≥14 kg)
Participants weighing \>=14 kg at screening received ELX100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 24 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
|
15 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
|
0 participants
|
—
|
PRIMARY outcome
Timeframe: From Day 1 up to Week 28Population: Safety set for Part B included all participants who received at least 1 dose of study drug in Part B. The safety and tolerability analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm.
Outcome measures
| Measure |
Part A: ELX/TEZ/IVA
n=75 Participants
Participants weighing \>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days.
|
Part B: ELX/TEZ/IVA (≥14 kg)
Participants weighing \>=14 kg at screening received ELX100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 24 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
|
74 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
|
2 participants
|
—
|
SECONDARY outcome
Timeframe: From Day 1 through Week 16Population: PK set included participants who received at least 1 dose of study drug. Here the "Number Analyzed" signifies those participants who were evaluable at specified time point.
Outcome measures
| Measure |
Part A: ELX/TEZ/IVA
n=16 Participants
Participants weighing \>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days.
|
Part B: ELX/TEZ/IVA (≥14 kg)
n=59 Participants
Participants weighing \>=14 kg at screening received ELX100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 24 weeks.
|
|---|---|---|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 4: ELX
|
3.13 micrograms per milliliter (mcg/ml)
Standard Deviation 1.90
|
3.56 micrograms per milliliter (mcg/ml)
Standard Deviation 2.19
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 4: IVA-M1
|
1.64 micrograms per milliliter (mcg/ml)
Standard Deviation 1.29
|
1.52 micrograms per milliliter (mcg/ml)
Standard Deviation 0.737
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 12: IVA-M1
|
1.19 micrograms per milliliter (mcg/ml)
Standard Deviation 0.602
|
1.45 micrograms per milliliter (mcg/ml)
Standard Deviation 0.907
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Day 15: ELX
|
2.91 micrograms per milliliter (mcg/ml)
Standard Deviation 1.38
|
3.87 micrograms per milliliter (mcg/ml)
Standard Deviation 2.75
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 12: ELX
|
2.48 micrograms per milliliter (mcg/ml)
Standard Deviation 1.65
|
3.49 micrograms per milliliter (mcg/ml)
Standard Deviation 2.22
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 16: ELX
|
3.01 micrograms per milliliter (mcg/ml)
Standard Deviation 1.11
|
3.18 micrograms per milliliter (mcg/ml)
Standard Deviation 2.19
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Day 15: ELX-M23
|
1.85 micrograms per milliliter (mcg/ml)
Standard Deviation 1.12
|
2.33 micrograms per milliliter (mcg/ml)
Standard Deviation 1.87
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Day 15: IVA-M1
|
1.47 micrograms per milliliter (mcg/ml)
Standard Deviation 0.969
|
1.67 micrograms per milliliter (mcg/ml)
Standard Deviation 0.961
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 4: ELX-M23
|
2.32 micrograms per milliliter (mcg/ml)
Standard Deviation 2.01
|
2.22 micrograms per milliliter (mcg/ml)
Standard Deviation 1.66
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 12: ELX-M23
|
1.48 micrograms per milliliter (mcg/ml)
Standard Deviation 1.27
|
2.17 micrograms per milliliter (mcg/ml)
Standard Deviation 1.69
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 16: ELX-M23
|
1.73 micrograms per milliliter (mcg/ml)
Standard Deviation 0.828
|
1.94 micrograms per milliliter (mcg/ml)
Standard Deviation 1.58
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Day 15: TEZ
|
1.73 micrograms per milliliter (mcg/ml)
Standard Deviation 0.666
|
2.14 micrograms per milliliter (mcg/ml)
Standard Deviation 1.72
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 4: TEZ
|
1.62 micrograms per milliliter (mcg/ml)
Standard Deviation 0.716
|
1.79 micrograms per milliliter (mcg/ml)
Standard Deviation 0.947
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 12: TEZ
|
1.60 micrograms per milliliter (mcg/ml)
Standard Deviation 0.951
|
1.76 micrograms per milliliter (mcg/ml)
Standard Deviation 1.10
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 16: TEZ
|
1.52 micrograms per milliliter (mcg/ml)
Standard Deviation 0.701
|
1.80 micrograms per milliliter (mcg/ml)
Standard Deviation 1.42
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Day 15: TEZ-M1
|
7.07 micrograms per milliliter (mcg/ml)
Standard Deviation 1.08
|
7.07 micrograms per milliliter (mcg/ml)
Standard Deviation 2.11
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 4: TEZ-M1
|
6.80 micrograms per milliliter (mcg/ml)
Standard Deviation 1.64
|
7.09 micrograms per milliliter (mcg/ml)
Standard Deviation 2.18
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 12: TEZ-M1
|
7.06 micrograms per milliliter (mcg/ml)
Standard Deviation 2.37
|
7.18 micrograms per milliliter (mcg/ml)
Standard Deviation 2.38
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 16: TEZ-M1
|
6.88 micrograms per milliliter (mcg/ml)
Standard Deviation 1.94
|
6.95 micrograms per milliliter (mcg/ml)
Standard Deviation 2.77
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Day 15: IVA
|
0.549 micrograms per milliliter (mcg/ml)
Standard Deviation 0.373
|
0.661 micrograms per milliliter (mcg/ml)
Standard Deviation 0.552
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 4: IVA
|
0.535 micrograms per milliliter (mcg/ml)
Standard Deviation 0.402
|
0.594 micrograms per milliliter (mcg/ml)
Standard Deviation 0.405
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 12: IVA
|
0.383 micrograms per milliliter (mcg/ml)
Standard Deviation 0.203
|
0.530 micrograms per milliliter (mcg/ml)
Standard Deviation 0.474
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 16: IVA
|
0.483 micrograms per milliliter (mcg/ml)
Standard Deviation 0.347
|
0.580 micrograms per milliliter (mcg/ml)
Standard Deviation 0.511
|
|
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites
Week 16: IVA-M1
|
1.24 micrograms per milliliter (mcg/ml)
Standard Deviation 0.673
|
1.51 micrograms per milliliter (mcg/ml)
Standard Deviation 0.911
|
SECONDARY outcome
Timeframe: From Baseline through Week 24Population: FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm.
Sweat samples were collected using an approved collection device.
Outcome measures
| Measure |
Part A: ELX/TEZ/IVA
n=75 Participants
Participants weighing \>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days.
|
Part B: ELX/TEZ/IVA (≥14 kg)
Participants weighing \>=14 kg at screening received ELX100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 24 weeks.
|
|---|---|---|
|
Part B: Absolute Change in Sweat Chloride (SwCl)
|
-57.9 millimole per liter (mmol/L)
Interval -61.3 to -54.6
|
—
|
SECONDARY outcome
Timeframe: From Baseline through Week 24Population: FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm.
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
Outcome measures
| Measure |
Part A: ELX/TEZ/IVA
n=63 Participants
Participants weighing \>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days.
|
Part B: ELX/TEZ/IVA (≥14 kg)
Participants weighing \>=14 kg at screening received ELX100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 24 weeks.
|
|---|---|---|
|
Part B: Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)
|
-0.83 index
Interval -1.01 to -0.66
|
—
|
Adverse Events
Part A: ELX/TEZ/IVA
Serious events: 0 serious events
Other events: 15 other events
Deaths: 0 deaths
Part B: ELX/TEZ/IVA
Serious events: 2 serious events
Other events: 71 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Part A: ELX/TEZ/IVA
n=18 participants at risk
Participants weighing \>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days.
|
Part B: ELX/TEZ/IVA
n=75 participants at risk
Participants weighing \>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing \>=10 kg to \<14 kg received ELX 80 mg qd/TEZ 40 mg qd/IVA 60 mg qAM and IVA 59.5 mg qPM in the treatment period for 24 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Anal incontinence
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
1.3%
1/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
1.3%
1/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Psychiatric disorders
Abnormal behaviour
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
1.3%
1/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Renal and urinary disorders
Urinary incontinence
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
1.3%
1/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
Other adverse events
| Measure |
Part A: ELX/TEZ/IVA
n=18 participants at risk
Participants weighing \>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days.
|
Part B: ELX/TEZ/IVA
n=75 participants at risk
Participants weighing \>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing \>=10 kg to \<14 kg received ELX 80 mg qd/TEZ 40 mg qd/IVA 60 mg qAM and IVA 59.5 mg qPM in the treatment period for 24 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal discomfort
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
6.7%
5/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Gastrointestinal disorders
Abdominal pain
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
5.3%
4/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Gastrointestinal disorders
Constipation
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
8.0%
6/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
6.7%
5/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
28.0%
21/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
General disorders
Pyrexia
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
34.7%
26/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Infections and infestations
COVID-19
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
18.7%
14/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
10.7%
8/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
8.0%
6/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Infections and infestations
Upper respiratory tract infection
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
14.7%
11/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Injury, poisoning and procedural complications
Scratch
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
0.00%
0/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
0.00%
0/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Investigations
Alanine aminotransferase increased
|
16.7%
3/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
10.7%
8/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Investigations
Aspartate aminotransferase increased
|
11.1%
2/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
5.3%
4/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Investigations
Gamma-glutamyltransferase increased
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
5.3%
4/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Investigations
SARS-CoV-2 test positive
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
9.3%
7/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
12.0%
9/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Metabolism and nutrition disorders
Hyperamylasaemia
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
0.00%
0/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Metabolism and nutrition disorders
Hyperlipasaemia
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
0.00%
0/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Nervous system disorders
Headache
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
9.3%
7/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Product Issues
Product taste abnormal
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
0.00%
0/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Psychiatric disorders
Irritability
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
5.3%
4/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
22.2%
4/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
61.3%
46/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
17.3%
13/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
4.0%
3/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
16.7%
3/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
33.3%
25/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
0.00%
0/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Skin and subcutaneous tissue disorders
Papule
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
0.00%
0/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
1.3%
1/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Skin and subcutaneous tissue disorders
Rash
|
11.1%
2/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
16.0%
12/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
1.3%
1/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
5.6%
1/18 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
1.3%
1/75 • Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)
The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. MedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place