Phase 3
Completed N=39
Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation
Source: ClinicalTrials.gov NCT01614470 ↗Enrolled (actual)
39
Serious AEs
12.6%
Results posted
Oct 2014
Primary outcomePrimary: Part 1: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8 — 76.3659; 79.3361; 8.1308; -5.8738 percent predicted of FEV1 — p=<0.0001
Summary
The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have a non-G551D cystic fibrosis transmembrane regulator (CFTR) gating mutation (any one of the following CFTR mutations: G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part 1: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8 |
76.3659; 79.3361; 8.1308; -5.8738 | <0.0001 sig |
| PRIMARY Part 2: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through 24 Weeks of Treatment (Week 36 Visit) |
74.8375; 13.5307 | — |
| SECONDARY Part 1: Change From Baseline in Body Mass Index (BMI) at Week 8 |
22.241; 22.527; 0.748; 0.043 | <0.0001 sig |
| SECONDARY Part 2: Change From Baseline in Body Mass Index (BMI) at 24 Weeks of Treatment (Week 36 Visit) |
22.222; 1.263 | — |
| SECONDARY Part 1: Change From Baseline in Sweat Chloride Through Week 8 |
93.37; 94.23; -55.82; -5.63 | <0.0001 sig |
| SECONDARY Part 2: Change From Baseline in Sweat Chloride Through 24 Weeks of Treatment (Week 36 Visit) |
92.03; -59.24 | — |
| SECONDARY Part 1: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8 |
70.61; 74.55; 12.31; -2.33 | 0.0004 sig |
| SECONDARY Part 2: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through 24 Weeks of Treatment (Week 36 Visit) |
71.30; 11.42 | — |
| SECONDARY Part 1: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
28; 31; 4; 7 | — |
| SECONDARY Part 2: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
30; 3 | — |
Eligibility Criteria
Inclusion Criteria
- Male or female with confirmed diagnosis of CF
- At least 1 allele of the following CFTR gating mutations: G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D
- Percent predicted forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent (%) predicted normal for age, sex, and height
- 6 years of age or older
- Minimum weight of 15 kilogram (kg) at screening
- Females of childbearing potential must not be pregnant
- Willing to comply with contraception requirements
Exclusion Criteria
- G551D-CFTR mutation on at least 1 allele
- History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the subject
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before the first dose of study drug
- History of solid organ or hematological transplantation
- History of alcohol, medication or illicit drug abuse within 1 year before the first dose of study drug
- Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days before screening
- Use of inhaled hypertonic saline treatment
- Use of any inhibitors or inducers of cytochrome (CYP) P450 3A
- Evidence of cataract or lens opacity at screening
Data sourced from ClinicalTrials.gov (NCT01614470). 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.