Trial Outcomes & Findings for Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation (NCT NCT05274269)
NCT ID: NCT05274269
Last Updated: 2024-08-01
Results Overview
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
307 participants
Primary outcome timeframe
From Baseline Through Week 24
Results posted on
2024-08-01
Participant Flow
This study was conducted in cystic fibrosis (CF) participants aged 6 years and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation.
Participant milestones
| Measure |
Placebo
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
Participants 6 to less than (\<) 12 years of age and weighing \<30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
102
|
205
|
|
Overall Study
COMPLETED
|
102
|
197
|
|
Overall Study
NOT COMPLETED
|
0
|
8
|
Reasons for withdrawal
| Measure |
Placebo
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
Participants 6 to less than (\<) 12 years of age and weighing \<30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
|---|---|---|
|
Overall Study
Adverse Event
|
0
|
3
|
|
Overall Study
Death
|
0
|
1
|
|
Overall Study
Other
|
0
|
2
|
|
Overall Study
Withdrawal of consent (not due to AE)
|
0
|
2
|
Baseline Characteristics
Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation
Baseline characteristics by cohort
| Measure |
Placebo
n=102 Participants
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
n=205 Participants
Participants 6 to \<12 years of age and weighing \<30kg at Day 1 received ELX 100 mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
Total
n=307 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Race/Ethnicity, Customized
Not Collected per Local Regulations
|
11 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
37 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Age, Continuous
|
33.9 years
STANDARD_DEVIATION 16.4 • n=5 Participants
|
33.3 years
STANDARD_DEVIATION 15.9 • n=7 Participants
|
33.5 years
STANDARD_DEVIATION 16.0 • n=5 Participants
|
|
Sex: Female, Male
Female
|
52 Participants
n=5 Participants
|
113 Participants
n=7 Participants
|
165 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
50 Participants
n=5 Participants
|
92 Participants
n=7 Participants
|
142 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
3 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
88 Participants
n=5 Participants
|
171 Participants
n=7 Participants
|
259 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
86 Participants
n=5 Participants
|
172 Participants
n=7 Participants
|
258 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White, Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
|
68.1 percent predicted FEV1
STANDARD_DEVIATION 18.1 • n=5 Participants
|
67.5 percent predicted FEV1
STANDARD_DEVIATION 17.6 • n=7 Participants
|
67.7 percent predicted FEV1
STANDARD_DEVIATION 17.7 • n=5 Participants
|
PRIMARY outcome
Timeframe: From Baseline Through Week 24Population: The Full Analysis Set (FAS) will include all randomized participants who carry the intended mutation and received at least 1 dose of study drug. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Outcome measures
| Measure |
Placebo
n=98 Participants
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
n=192 Participants
Participants 6 to \<12 years of age and weighing \<30kg at Day 1 received ELX 100 mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
|---|---|---|
|
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
|
-0.4 percent predicted FEV1
Interval -2.0 to 1.3
|
8.9 percent predicted FEV1
Interval 7.7 to 10.0
|
SECONDARY outcome
Timeframe: From Baseline Through Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure.
Sweat samples were collected using an approved collection device.
Outcome measures
| Measure |
Placebo
n=100 Participants
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
n=200 Participants
Participants 6 to \<12 years of age and weighing \<30kg at Day 1 received ELX 100 mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
|---|---|---|
|
Absolute Change in Sweat Chloride (SwCl)
|
0.5 millimole per liter (mmol/L)
Interval -2.6 to 3.6
|
-27.8 millimole per liter (mmol/L)
Interval -30.0 to -25.6
|
SECONDARY outcome
Timeframe: From Baseline Through Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure.
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Outcome measures
| Measure |
Placebo
n=102 Participants
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
n=202 Participants
Participants 6 to \<12 years of age and weighing \<30kg at Day 1 received ELX 100 mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
|---|---|---|
|
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
|
-2.0 units on a scale
Interval -5.2 to 1.3
|
17.5 units on a scale
Interval 15.2 to 19.8
|
SECONDARY outcome
Timeframe: From Baseline at Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure.
BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2).
Outcome measures
| Measure |
Placebo
n=102 Participants
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
n=196 Participants
Participants 6 to \<12 years of age and weighing \<30kg at Day 1 received ELX 100 mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
|---|---|---|
|
Absolute Change in Body Mass Index (BMI)
|
0.35 kg/m^2
Interval 0.16 to 0.53
|
0.81 kg/m^2
Interval 0.68 to 0.94
|
SECONDARY outcome
Timeframe: From Baseline at Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure.
Outcome measures
| Measure |
Placebo
n=102 Participants
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
n=196 Participants
Participants 6 to \<12 years of age and weighing \<30kg at Day 1 received ELX 100 mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
|---|---|---|
|
Absolute Change in Weight
|
1.2 kilogram (kg)
Interval 0.6 to 1.7
|
2.4 kilogram (kg)
Interval 2.1 to 2.8
|
SECONDARY outcome
Timeframe: From Baseline Through Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure.
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Outcome measures
| Measure |
Placebo
n=102 Participants
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
n=205 Participants
Participants 6 to \<12 years of age and weighing \<30kg at Day 1 received ELX 100 mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
|---|---|---|
|
Number of Pulmonary Exacerbations (PEx)
|
40 PEx events
|
21 PEx events
|
SECONDARY outcome
Timeframe: Day 1 up to Week 28Population: Safety Set was defined as all participants who received at least 1 dose of study drug.
Outcome measures
| Measure |
Placebo
n=102 Participants
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
n=205 Participants
Participants 6 to \<12 years of age and weighing \<30kg at Day 1 received ELX 100 mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
|---|---|---|
|
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with TEAEs
|
97 Participants
|
193 Participants
|
|
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with SAEs
|
15 Participants
|
18 Participants
|
Adverse Events
Placebo
Serious events: 15 serious events
Other events: 86 other events
Deaths: 0 deaths
ELX/TEZ/IVA
Serious events: 18 serious events
Other events: 164 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Placebo
n=102 participants at risk
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
n=205 participants at risk
Participants 6 to \<12 years of age and weighing \<30 kg at Day 1 received ELX 100mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
|---|---|---|
|
Cardiac disorders
Stress cardiomyopathy
|
0.98%
1/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.00%
0/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Pancreatitis
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Subileus
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
General disorders
Drug intolerance
|
0.98%
1/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.00%
0/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Hepatobiliary disorders
Cholecystitis acute
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Bronchopulmonary aspergillosis allergic
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.98%
2/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Hepatitis viral
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
12.7%
13/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
2.4%
5/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Pseudomonas infection
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Vestibular neuronitis
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Polyarthritis
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lung adenocarcinoma
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Psychiatric disorders
Anxiety disorder
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Psychiatric disorders
Post-traumatic stress disorder
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Renal and urinary disorders
Renal colic
|
0.98%
1/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.00%
0/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
0.49%
1/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
Other adverse events
| Measure |
Placebo
n=102 participants at risk
Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
|
ELX/TEZ/IVA
n=205 participants at risk
Participants 6 to \<12 years of age and weighing \<30 kg at Day 1 received ELX 100mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
12.7%
13/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
8.3%
17/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
6.9%
7/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
4.9%
10/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Constipation
|
3.9%
4/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
7.3%
15/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Diarrhoea
|
9.8%
10/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
12.7%
26/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Vomiting
|
6.9%
7/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
7.3%
15/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
General disorders
Pyrexia
|
13.7%
14/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
13.2%
27/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
COVID-19
|
9.8%
10/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
9.3%
19/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
35.3%
36/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
13.7%
28/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Influenza
|
2.0%
2/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
8.8%
18/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Nasopharyngitis
|
19.6%
20/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
20.5%
42/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Rhinitis
|
5.9%
6/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
9.8%
20/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Upper respiratory tract infection
|
9.8%
10/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
8.3%
17/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Nervous system disorders
Headache
|
12.7%
13/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
18.0%
37/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
25.5%
26/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
17.6%
36/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
5.9%
6/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
5.9%
12/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
7.8%
8/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
2.9%
6/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
9.8%
10/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
8.3%
17/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
5.9%
6/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
2.9%
6/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
12.7%
13/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
9.8%
20/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.98%
1/102 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
22.0%
45/205 • Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place