Phase 3
Completed N=161
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation
Source: ClinicalTrials.gov NCT00909532 ↗Enrolled (actual)
161
Serious AEs
32.9%
Results posted
Aug 2012
Primary outcomePrimary: Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24 — -0.2; 10.4 percent of predicted volume (L) — p=<0.0001
Summary
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24 |
-0.2; 10.4 | <0.0001 sig |
| SECONDARY Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48 |
-0.4; 10.1 | <0.0001 sig |
| SECONDARY Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled) |
-2.1; 6.0; -2.7; 6.0 | <0.0001 sig |
| SECONDARY Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48 |
-0.8; -48.7; -0.6; -48.7 | <0.0001 sig |
| SECONDARY Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48 |
0.97; 0.98; 0.87; 0.89; 0.72; 0.83 | 0.0016 sig |
| SECONDARY Absolute Change From Baseline in Weight at Week 24 and Week 48 |
0.2; 3.0; 0.4; 3.1 | <0.0001 sig |
Eligibility Criteria
Inclusion Criteria
- Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
- Forced expiratory volume in 1 second (FEV1) of 40% to 90% (inclusive) of predicted normal for age, gender, and height at Screening.
- No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
- Willing to use highly effective birth control methods during the study
Exclusion Criteria
- History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
- Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 of the study
- History of alcohol, medication or illicit drug abuse within one year prior to Day 1
- Abnormal liver function ≥ 3x the upper limit of normal
- Abnormal renal function at Screening
- History of solid organ or hematological transplantation
- Pregnant, planning a pregnancy, breast-feeding, or unwilling to follow contraception requirements
- Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening
- Use of inhaled hypertonic saline treatment
- Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP 3A4)
Data sourced from ClinicalTrials.gov (NCT00909532). 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.