Phase 2
Completed N=21
Safety Study of Ivacaftor in Subjects With Cystic Fibrosis
Source: ClinicalTrials.gov NCT00457821 ↗Enrolled (actual)
21
Serious AEs
1.8%
Results posted
Oct 2012
Primary outcomePrimary: Number of Subjects With Adverse Events (Combined Part 1 and Part 2) — 7; 26; 3; 13 participants
Summary
The purpose of this study was to evaluate the safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a 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 Number of Subjects With Adverse Events (Combined Part 1 and Part 2) |
7; 26; 3; 13; 0; 1 | — |
| PRIMARY Number of Adverse Events (Combined Part 1 and Part 2) |
32; 179; 11; 40; 0; 2 | — |
| SECONDARY Change From Baseline in Nasal Potential Difference (Combined Part 1 and Part 2) |
-0.3; -1.4; -4.4; -4.6; -7.6; -0.5 | <0.05 sig |
| SECONDARY Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second [FEV1] (Combined Part 1 and Part 2) |
0.5; 2.7; 5.1; 6.9; 8.4; 2.1 | <0.05 sig |
| SECONDARY Change From Baseline in the Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score (Part 2 Only)(Respiratory Domain Score) |
70.8; 68.8; 73.0; 2.8; 6.3; 5.6 | — |
| SECONDARY Change From Baseline in Maximum Sweat Chloride Concentration (Combined Part 1 and Part 2) |
2.0; -33.8; -42.0; -46.0; -27.1; 4.9 | <0.05 sig |
Eligibility Criteria
Inclusion Criteria
- Weighing at least 40 kg
- Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
- Forced expiratory volume in 1 second (FEV1) of at least 40% of predicted normal for age, gender, and height
- Willing to remain on stable medication regimen for the duration of study participation
- No significant clinical laboratory abnormalities, not pregnant, and willing to use at least 2 highly effective birth control methods during Part 1 and 1 highly effective birth control method during Part 2 of the study
- No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
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
- Ongoing acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 14 days 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
- History of abnormal renal function (creatinine clearance < 50 mL/min using Cockcroft-Gault equation)
- History of solid organ or hematological transplantation
- Pregnant or breast-feeding (for women)
- Ongoing participation in another therapeutic clinical trial, or prior participation in an investigational drug study without appropriate washout
- Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP3A4)
Data sourced from ClinicalTrials.gov (NCT00457821). 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.