Phase 3
Completed N=125
Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
Source: ClinicalTrials.gov NCT01707290 ↗Enrolled (actual)
125
Serious AEs
22.4%
Results posted
May 2017
Primary outcomePrimary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm — 117; 27 participants
◆ Published Evidence
Highly cited
224citations · ~20 / year
Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial.
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).
Linked Publications (2)
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Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial.
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Long-Term Ivacaftor in People Aged 6 Years and Older with Cystic Fibrosis with Ivacaftor-Responsive Mutations.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm |
117; 27 | — |
| 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 |
71.8; 78.3; 62.8; 3.8; 4.4; 4 | — |
| SECONDARY Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 |
23.75; 23.15; 24.81; 0.06; 0.16; 0.07 | — |
| SECONDARY Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 |
60.9; 80.2; 55.7; -19.3; -38.4; -4.5 | — |
| 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 |
68.6; 72.4; 65.9; 5.5; 4.5; 13.2 | — |
| SECONDARY Number of Pulmonary Exacerbations Events |
47; 30; 6 | — |
| SECONDARY Number of Participants With Serious Adverse Events (SAEs) in Observational Arm |
1 | — |
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT01707290) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.