Cystic Fibrosis
Conditions
Brief summary
The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in participants with cystic fibrosis (CF) from Studies 110 (NCT01614457), 111 (NCT01614470), and 113 (NCT01685801).
Detailed description
Ivacaftor is the first Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator to show an improvement in CFTR function and clinical benefit in participants with CF. Results from Phase 3 studies (NCT00909532 \[Study 102\] and NCT00909727 \[Study 103\]) showed that ivacaftor is effective in the treatment of participants with CF who have the G551D-CFTR mutation, as evidenced by sustained improvements in CFTR channel function (measured by reduction in sweat chloride concentration) and corresponding substantial, durable improvements in lung function, pulmonary exacerbations, respiratory symptoms, and weight gain. Ivacaftor was also well tolerated, as evidenced by the rates and reasons for premature discontinuation and results of safety assessments. Ivacaftor (Trade Name Kalydeco; 150 mg tablets) was initially approved in the United States for the treatment of CF in participants 6 years of age and older who have a G551D mutation in the CFTR gene.
Interventions
DRUGIvacaftor
150 mg tablet, oral use, every 12 hours (q12h)
Sponsors
Cystic Fibrosis Foundation
Vertex Pharmaceuticals Incorporated
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participants from Study 110 or Study 111 entering the ivacaftor arm must have completed the assigned study drug treatment duration in the previous study. * Participants from Study 113 entering the ivacaftor arm must have completed all study related treatments through the Follow-up Visit and met the Study 113 responder criteria during the previous study. * Participants entering the observational arm must have completed at least 4 weeks of study drug treatment in their previous study (Study 110 or Study 111), must have completed the previous study but do not wish to enroll in the ivacaftor arm, or must have completed the previous study but do not meet the inclusion criteria of the ivacaftor arm. * Participants of childbearing potential entering the ivacaftor arm must not be pregnant. * Participants entering the ivacaftor arm must be willing to comply with contraception requirements.
Exclusion criteria
(Ivacaftor Arm Only): * History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the Participant. * Use of moderate or strong inhibitors or inducers of cytochrome P450 (CYP) 3A. * Evidence of cataract or lens opacity at or before the Day 1 Visit.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm | Day 1 up to Week 108 (Study 112) | AE: any untoward medical occurrence in a participants during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. TEAEs were defined as adverse events with start date or increased severity on and after the first dose of study drug through Week 108. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112) | BMI was defined as weight in kg divided by height in m\^2. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113. |
| Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Baseline, Week 2, 24, 48 and 104 (Study 112) | Sweat samples were collected using an approved collection device. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113. |
| Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 6 to 17 years and for female participants aged 6 to 15 years. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113. |
| Number of Pulmonary Exacerbations Events | Through Week 104 (Study 112) | Pulmonary exacerbation events include those events which require treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. The number of events were reported. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113. |
| Number of Participants With Serious Adverse Events (SAEs) in Observational Arm | up to 2 years (Study 112) | SAE was defined as a medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. |
| Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112) | The CFQ-R is a validated participant reported outcome measuring health related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health related quality of life. Baseline was defined as the most recent measurement before intake of the first dose of study drug (ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113. |
Countries
Belgium, France, United Kingdom, United States
Participant flow
Pre-assignment details
The study consisted of 2 arms: Ivacaftor arm and Observational arm. The Ivacaftor arm enrolled subjects from Study VX11-770-110 (NCT01614457), Study VX12-770-111 (NCT01614470) and Study VX12-770-113 (NCT01685801). The Observational arm enrolled subjects from Study VX11-770-110 (NCT01614457) and Study VX12-770-111 (NCT01614470).
Participants by arm
| Arm | Count |
|---|---|
| Ivacaftor Arm Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet, q12h in the previous study VX11-770-110 (Study 110; NCT01614457), VX12-770-111 (Study 111; NCT01614470) or VX12-770-113 (Study 113; NCT01685801); received Ivacaftor 150 mg tablet orally q12h in this VX12-770-112 (Study 112; NCT01707290) up to 104 weeks. | 121 |
| Observational Arm Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet q12h in the previous Study 110 (NCT01614457) or Study 111 (NCT01614470), were observed (did not receive study drug) in this Study 112 (NCT01707290) for up to 2 years. | 4 |
| Total | 125 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse event (AE) | 1 | 0 |
| Overall Study | Commercial drug available | 60 | 1 |
| Overall Study | Lost to Follow-up | 3 | 0 |
| Overall Study | Other unspecified | 17 | 0 |
| Overall Study | Physician Decision | 1 | 0 |
| Overall Study | Withdrawal of Consent (Not Due to AE) | 4 | 0 |
Baseline characteristics
| Characteristic | Ivacaftor Arm | Observational Arm | Total |
|---|---|---|---|
| Age, Customized 12 to 17 years | 11 participants | 1 participants | 12 participants |
| Age, Customized 18 years and over | 89 participants | 2 participants | 91 participants |
| Age, Customized 6 to 11 years | 21 participants | 1 participants | 22 participants |
| Sex: Female, Male Female | 61 Participants | 3 Participants | 64 Participants |
| Sex: Female, Male Male | 60 Participants | 1 Participants | 61 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 117 / 121 | 0 / 0 |
| serious Total, serious adverse events | 27 / 121 | 1 / 4 |
Outcome results
Primary
Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm
AE: any untoward medical occurrence in a participants during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. TEAEs were defined as adverse events with start date or increased severity on and after the first dose of study drug through Week 108.
Time frame: Day 1 up to Week 108 (Study 112)
Population: Safety Set included all participants who received at least 1 dose of study drug (Ivacaftor).
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Ivacaftor Arm | Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm | Participants with TEAEs | 117 participants |
| Ivacaftor Arm | Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm | Participants with SAEs | 27 participants |
Secondary
Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104
BMI was defined as weight in kg divided by height in m\^2. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
Time frame: Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)
Population: FAS included all participants who received at least 1 dose of study drug (Ivacaftor). Here, Number analyzed signifies those participants who were evaluable for this measure at the specified time point for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ivacaftor Arm | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 84 | 0.72 kilogram per square meter (kg/m^2) | Standard Deviation 1.51 |
| Ivacaftor Arm | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 36 | 0.62 kilogram per square meter (kg/m^2) | Standard Deviation 1.04 |
| Ivacaftor Arm | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 24 | 0.43 kilogram per square meter (kg/m^2) | Standard Deviation 0.92 |
| Ivacaftor Arm | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 12 | 0.32 kilogram per square meter (kg/m^2) | Standard Deviation 0.59 |
| Ivacaftor Arm | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 48 | 0.54 kilogram per square meter (kg/m^2) | Standard Deviation 1.39 |
| Ivacaftor Arm | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 104 | 0.42 kilogram per square meter (kg/m^2) | Standard Deviation 1.49 |
| Ivacaftor Arm | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 2 | 0.06 kilogram per square meter (kg/m^2) | Standard Deviation 0.43 |
| Ivacaftor Arm | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 60 | 0.72 kilogram per square meter (kg/m^2) | Standard Deviation 1.32 |
| Ivacaftor Arm | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Baseline | 23.75 kilogram per square meter (kg/m^2) | Standard Deviation 5.63 |
| Ivacaftor Arm | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 96 | 0.49 kilogram per square meter (kg/m^2) | Standard Deviation 1.44 |
| Ivacaftor Arm | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 72 | 1.05 kilogram per square meter (kg/m^2) | Standard Deviation 1.73 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Baseline | 23.15 kilogram per square meter (kg/m^2) | Standard Deviation 5.28 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 12 | 0.39 kilogram per square meter (kg/m^2) | Standard Deviation 0.62 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 84 | 0.33 kilogram per square meter (kg/m^2) | Standard Deviation 1.42 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 96 | 0.1 kilogram per square meter (kg/m^2) | Standard Deviation 1.49 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 104 | 0.16 kilogram per square meter (kg/m^2) | Standard Deviation 1.37 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 24 | 0.65 kilogram per square meter (kg/m^2) | Standard Deviation 0.96 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 72 | 0.45 kilogram per square meter (kg/m^2) | Standard Deviation 1.34 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 36 | 0.64 kilogram per square meter (kg/m^2) | Standard Deviation 1.07 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 48 | 0.58 kilogram per square meter (kg/m^2) | Standard Deviation 1.22 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 60 | 0.25 kilogram per square meter (kg/m^2) | Standard Deviation 1.09 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 2 | 0.16 kilogram per square meter (kg/m^2) | Standard Deviation 0.47 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 104 | 0.88 kilogram per square meter (kg/m^2) | Standard Deviation 1.39 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Baseline | 24.81 kilogram per square meter (kg/m^2) | Standard Deviation 5.76 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 12 | 0.22 kilogram per square meter (kg/m^2) | Standard Deviation 0.6 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 24 | 0.17 kilogram per square meter (kg/m^2) | Standard Deviation 0.91 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 36 | 0.02 kilogram per square meter (kg/m^2) | Standard Deviation 1.27 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 48 | 0.28 kilogram per square meter (kg/m^2) | Standard Deviation 1.26 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 60 | 0.38 kilogram per square meter (kg/m^2) | Standard Deviation 1.22 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 72 | 0.22 kilogram per square meter (kg/m^2) | Standard Deviation 1.22 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 84 | 0.23 kilogram per square meter (kg/m^2) | Standard Deviation 0.92 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 96 | 0.52 kilogram per square meter (kg/m^2) | Standard Deviation 1.28 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 2 | 0.07 kilogram per square meter (kg/m^2) | Standard Deviation 0.43 |
Secondary
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 6 to 17 years and for female participants aged 6 to 15 years. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
Time frame: Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)
Population: Full Analysis Set (FAS) included all participants who received at least 1 dose of study drug (Ivacaftor). Here, Number Analyzed signifies those participants who were evaluable for this measure at the specified time point for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ivacaftor Arm | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 24 | 4.5 Percent predicted of FEV1 | Standard Deviation 8.4 |
| Ivacaftor Arm | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 36 | 4.4 Percent predicted of FEV1 | Standard Deviation 7.1 |
| Ivacaftor Arm | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Baseline | 71.8 Percent predicted of FEV1 | Standard Deviation 20.4 |
| Ivacaftor Arm | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 2 | 3.8 Percent predicted of FEV1 | Standard Deviation 8 |
| Ivacaftor Arm | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 12 | 5.4 Percent predicted of FEV1 | Standard Deviation 8.9 |
| Ivacaftor Arm | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 48 | 4.4 Percent predicted of FEV1 | Standard Deviation 7.5 |
| Ivacaftor Arm | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 60 | 4.5 Percent predicted of FEV1 | Standard Deviation 8.4 |
| Ivacaftor Arm | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 72 | 3.6 Percent predicted of FEV1 | Standard Deviation 8.9 |
| Ivacaftor Arm | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 84 | 5.4 Percent predicted of FEV1 | Standard Deviation 13.5 |
| Ivacaftor Arm | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 96 | 4.1 Percent predicted of FEV1 | Standard Deviation 14.6 |
| Ivacaftor Arm | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 104 | 4.6 Percent predicted of FEV1 | Standard Deviation 13.2 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 84 | 7.7 Percent predicted of FEV1 | Standard Deviation 7.6 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 24 | 7.1 Percent predicted of FEV1 | Standard Deviation 8.3 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 36 | 7.5 Percent predicted of FEV1 | Standard Deviation 8.2 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 48 | 6.4 Percent predicted of FEV1 | Standard Deviation 10.7 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 104 | 4.9 Percent predicted of FEV1 | Standard Deviation 5.8 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 60 | 8.2 Percent predicted of FEV1 | Standard Deviation 8.3 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 96 | 9.9 Percent predicted of FEV1 | Standard Deviation 6.4 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 72 | 7.8 Percent predicted of FEV1 | Standard Deviation 9.2 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Baseline | 78.3 Percent predicted of FEV1 | Standard Deviation 21.1 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 2 | 4.4 Percent predicted of FEV1 | Standard Deviation 7.7 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 12 | 5.5 Percent predicted of FEV1 | Standard Deviation 8.3 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 104 | 6.3 Percent predicted of FEV1 | Standard Deviation 10 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 2 | 4 Percent predicted of FEV1 | Standard Deviation 7.2 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 24 | 4.6 Percent predicted of FEV1 | Standard Deviation 8.5 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 96 | 5.9 Percent predicted of FEV1 | Standard Deviation 8.1 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 36 | 4.9 Percent predicted of FEV1 | Standard Deviation 8.2 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 84 | 4.8 Percent predicted of FEV1 | Standard Deviation 7.2 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 72 | 5.9 Percent predicted of FEV1 | Standard Deviation 9.2 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 48 | 4.9 Percent predicted of FEV1 | Standard Deviation 9 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 12 | 4.9 Percent predicted of FEV1 | Standard Deviation 6.7 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Baseline | 62.8 Percent predicted of FEV1 | Standard Deviation 22.2 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 | Change at Week 60 | 4.5 Percent predicted of FEV1 | Standard Deviation 8.8 |
Secondary
Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104
The CFQ-R is a validated participant reported outcome measuring health related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health related quality of life. Baseline was defined as the most recent measurement before intake of the first dose of study drug (ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
Time frame: Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)
Population: FAS included all participants who received at least 1 dose of study drug (ivacaftor). Here, Number Analyzed signifies those participants who were evaluable for this measure at the specified time point for each arm respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ivacaftor Arm | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 60 | 7.1 units on a scale | Standard Deviation 18.6 |
| Ivacaftor Arm | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 24 | 6.7 units on a scale | Standard Deviation 19 |
| Ivacaftor Arm | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 84 | 10.5 units on a scale | Standard Deviation 19.5 |
| Ivacaftor Arm | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 48 | 9.5 units on a scale | Standard Deviation 17.6 |
| Ivacaftor Arm | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 36 | 12.1 units on a scale | Standard Deviation 19.3 |
| Ivacaftor Arm | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 96 | 8.8 units on a scale | Standard Deviation 21.1 |
| Ivacaftor Arm | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 2 | 5.5 units on a scale | Standard Deviation 17.9 |
| Ivacaftor Arm | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 104 | 9.2 units on a scale | Standard Deviation 14 |
| Ivacaftor Arm | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 72 | 10.4 units on a scale | Standard Deviation 22.2 |
| Ivacaftor Arm | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 12 | 11.6 units on a scale | Standard Deviation 18 |
| Ivacaftor Arm | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Baseline | 68.6 units on a scale | Standard Deviation 21.6 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 72 | 5.6 units on a scale | Standard Deviation 11.8 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Baseline | 72.4 units on a scale | Standard Deviation 20.3 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 2 | 4.5 units on a scale | Standard Deviation 17.5 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 12 | 5.7 units on a scale | Standard Deviation 18.5 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 24 | 3.4 units on a scale | Standard Deviation 18.7 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 36 | 5.7 units on a scale | Standard Deviation 13.8 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 48 | 4.4 units on a scale | Standard Deviation 13.2 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 60 | 4.4 units on a scale | Standard Deviation 10.9 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 84 | 8.8 units on a scale | Standard Deviation 14.1 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 96 | 0 units on a scale | Standard Deviation 9.5 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 104 | 1.9 units on a scale | Standard Deviation 8.8 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 104 | 19.9 units on a scale | Standard Deviation 16 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 60 | 8.6 units on a scale | Standard Deviation 19.3 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 2 | 13.2 units on a scale | Standard Deviation 16.2 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 72 | 8.6 units on a scale | Standard Deviation 19 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 96 | 9.4 units on a scale | Standard Deviation 14.7 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 84 | 4.9 units on a scale | Standard Deviation 14.6 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 36 | 7.9 units on a scale | Standard Deviation 20.5 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 24 | 8.5 units on a scale | Standard Deviation 14.9 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Baseline | 65.9 units on a scale | Standard Deviation 18.8 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 48 | 11.4 units on a scale | Standard Deviation 15.7 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 | Change at Week 12 | 13.5 units on a scale | Standard Deviation 20 |
Secondary
Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104
Sweat samples were collected using an approved collection device. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
Time frame: Baseline, Week 2, 24, 48 and 104 (Study 112)
Population: FAS included all participants who received at least 1 dose of study drug (Ivacaftor). Here, Number of participants analyzed signifies those participants who were evaluable for this outcome and Number Analyzed signifies those participants who were evaluable for this measure at the specified time point for each arm respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ivacaftor Arm | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 2 | -19.3 millimole per liter (mmol/L) | Standard Deviation 10.7 |
| Ivacaftor Arm | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Baseline | 60.9 millimole per liter (mmol/L) | Standard Deviation 19.4 |
| Ivacaftor Arm | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 24 | -13 millimole per liter (mmol/L) | Standard Deviation 18.4 |
| Ivacaftor Arm | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 48 | -13.5 millimole per liter (mmol/L) | Standard Deviation 16.2 |
| Ivacaftor Arm | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 104 | -13.7 millimole per liter (mmol/L) | Standard Deviation 22.9 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 24 | -39.2 millimole per liter (mmol/L) | Standard Deviation 27 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 104 | -32.9 millimole per liter (mmol/L) | Standard Deviation 26.8 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 48 | -40.6 millimole per liter (mmol/L) | Standard Deviation 26.1 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Baseline | 80.2 millimole per liter (mmol/L) | Standard Deviation 22.8 |
| Ivacaftor Arm: Study 111 | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 2 | -38.4 millimole per liter (mmol/L) | Standard Deviation 27.5 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Baseline | 55.7 millimole per liter (mmol/L) | Standard Deviation 22.2 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 2 | -4.5 millimole per liter (mmol/L) | Standard Deviation 10.9 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 48 | -1.9 millimole per liter (mmol/L) | Standard Deviation 17.8 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 24 | 1.1 millimole per liter (mmol/L) | Standard Deviation 15.8 |
| Ivacaftor Arm: Study 113 | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 | Change at Week 104 | 5.4 millimole per liter (mmol/L) | Standard Deviation 18.1 |
Secondary
Number of Participants With Serious Adverse Events (SAEs) in Observational Arm
SAE was defined as a medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event.
Time frame: up to 2 years (Study 112)
Population: Analysis population included all participants who were included in the observational arm.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ivacaftor Arm | Number of Participants With Serious Adverse Events (SAEs) in Observational Arm | 1 participants |
Secondary
Number of Pulmonary Exacerbations Events
Pulmonary exacerbation events include those events which require treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. The number of events were reported. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
Time frame: Through Week 104 (Study 112)
Population: FAS included all participants who received at least 1 dose of study drug (Ivacaftor).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ivacaftor Arm | Number of Pulmonary Exacerbations Events | 47 pulmonary exacerbation events |
| Ivacaftor Arm: Study 111 | Number of Pulmonary Exacerbations Events | 30 pulmonary exacerbation events |
| Ivacaftor Arm: Study 113 | Number of Pulmonary Exacerbations Events | 6 pulmonary exacerbation events |