Phase 3
Completed N=69
Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT)
Source: ClinicalTrials.gov NCT01614457 ↗Enrolled (actual)
69
Serious AEs
14.5%
Results posted
Feb 2015
Primary outcomePrimary: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24 — 2.5724; 0.4611 percent predicted of FEV1 — p=0.1979
Summary
The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have the R117H-CFTR mutation.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24 |
2.5724; 0.4611 | 0.1979 |
| SECONDARY Change From Baseline in Body Mass Index (BMI) at Week 24 |
0.4910; 0.2284 | 0.7780 |
| SECONDARY Change From Baseline in Sweat Chloride Through Week 24 |
-26.2771; -2.3078 | <0.0001 sig |
| SECONDARY Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24 |
7.5585; -0.8289 | 0.0091 sig |
| SECONDARY Time to First Pulmonary Exacerbation |
3; 1; 4; 2; 2; 6 | 0.8556 |
| SECONDARY Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
32; 35; 4; 6 | — |
Eligibility Criteria
Inclusion Criteria
- Male or female with confirmed diagnosis of CF
- Must have at least 1 allele of the R117H CFTR mutation
- Percent predicted forced expiratory volume in 1 second (FEV1) 40 percent (%) to 90% (for subjects aged 12 years or older) or 40% to 105% (for subjects aged 6 to 11 years) 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
- CFTR gene mutation leading to CFTR channel with gating defect (that is, any 1 of the following mutations: G551D, G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D)
- 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
- Abnormal liver function, at screening, defined as greater than or equal to (>=) 3 time upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), total bilirubin
- Colonization with organisms associated with a more rapid decline in pulmonary status (for example, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus) at screening
- 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
- Any "non-CF-related" illness within 2 weeks before Day 1 (first dose of study drug). "Illness" was defined as an acute (serious or non-serious) condition (for example, gastroenteritis)
- Use of any inhibitors or inducers of cytochrome (CYP) P450 3A
Data sourced from ClinicalTrials.gov (NCT01614457). 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.