At a glance
ClinicalIndex Comparison Record- ✓Confirmed cystic fibrosis diagnosis with G551D mutation in at least one allele
- ✓FEV1 at least 40% of predicted normal for age, gender, and height
- ✓Body weight at least 40 kg
- ✓Willing to remain on stable medication regimen throughout study participation
- ✕Ongoing acute respiratory infection, pulmonary exacerbation, or changes in pulmonary therapy within 14 days before Day 1
- ✕Abnormal liver function ≥3× upper limit of normal
- ✕Abnormal renal function (creatinine clearance <50 mL/min by Cockcroft-Gault)
- ✕Prior solid organ or hematological transplantation
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study of VX-770 to Evaluate Safety, Pharmacokinetics, and Biomarkers of CFTR Activity in Cystic Fibrosis (CF) Subjects With Genotype G551D
In Brief
A Phase 2 clinical trial evaluating Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg, and 2 other interventions for Cystic Fibrosis. Completed, enrolled 39 participants across 15 sites in 3 countries.
Detailed 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.
Study Details
Timeline
Interventions
25 mg or 75 mg q12h for a total of 28 days (Part 1)
75 mg or 150 mg q12h for a total of 28 days (Part 1)
150 mg or 250 mg of ivacaftor q12h for 28 days (Part 2)
Given q12h for 28 days each in Part 1 and Part 2 of the study