Overview

This trial is active, not recruiting.

Condition cystic fibrosis
Treatments vx-661/ ivacaftor, ivacaftor, vx-661/ ivacaftor placebo, ivacaftor placebo
Phase phase 3
Sponsor Vertex Pharmaceuticals Incorporated
Start date January 2015
End date December 2016
Trial size 490 participants
Trial identifier NCT02347657, VX14-661-106

Summary

This is a Phase 3, randomized, double blind, placebo controlled, parallel group, multicenter study in people with CF who are homozygous for the F508del CFTR mutation.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator)
Primary purpose treatment
Arm
(Experimental)
100-mg VX-661/150-mg ivacaftor fixed-dose combination film coated tablet for oral administration (morning dose) 150-mg ivacaftor film coated tablet for oral administration (evening dose)
vx-661/ ivacaftor
ivacaftor
(Placebo Comparator)
0-mg film coated matching placebo tablets for oral administration in morning and evening
vx-661/ ivacaftor placebo
ivacaftor placebo

Primary Outcomes

Measure
Absolute change in percent predicted forced expiratory volume in 1 second (FEV1)
time frame: baseline through Week 24

Secondary Outcomes

Measure
Relative change in percent predicted FEV1
time frame: baseline through Week 24
Number of pulmonary exacerbations
time frame: through Week 24
Absolute change in body mass index (BMI)
time frame: baseline at Week 24
Absolute change in Cystic Fibrosis Questionnaire - Revised (CFQ-R) respiratory domain score
time frame: baseline through Week 24
Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values standard 12-lead electrocardiograms (ECGs), vital signs, and pulse oximetry
time frame: Screening through 4 weeks after receiving last dose
Time-to-first pulmonary exacerbation
time frame: through Week 24
Absolute change in sweat chloride
time frame: baseline through Week 24
Absolute change in BMI z-score (in subjects <20 years of age at time of screening)
time frame: baseline at Week 24
Absolute change in body weight
time frame: baseline at Week 24
Pharmacokinetic parameters; Area under the plasma concentration versus time curve (AUC) of VX-661, M1-661, M2-661, ivacaftor, and M1-ivacaftor, as determined by population analysis.
time frame: Day 1 through Week 24
Pharmacokinetic parameters; Peak Plasma Concentration (Cmax) of VX-661, M1-661, M2-661, ivacaftor, and M1-ivacaftor, as determined by population analysis.
time frame: Day 1 through Week 24

Eligibility Criteria

Male or female participants at least 12 years old.

Inclusion Criteria: - Homozygous for the F508del CFTR mutation, genotype to be confirmed at the Screening Visit - Confirmed diagnosis of CF defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis - FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height during screening - Stable CF disease as judged by the investigator - Willing to remain on a stable CF medication regimen through Week 24 or, if applicable, the Safety Follow up Visit Exclusion Criteria: - History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. - An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug) - Pregnant or nursing females (females of childbearing potential must have a negative pregnancy test at Screening and Day 1) - Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements

Additional Information

Official title A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Description This is a Phase 3, randomized, double-blind, placebo-controlled, parallel-group, multicenter study in people with CF who are homozygous for the F508del-CFTR mutation. This study is designed to evaluate the efficacy and safety of VX-661 in combination with ivacaftor. The active treatment regimen will be comprised of a morning dose of a fixed-dose combination tablet of 100 mg VX-661/150 mg ivacaftor once daily (qd) and an evening dose of ivacaftor 150 mg to be taken approximately 12 hours after the morning dose. The placebo regimen will be visually matched tablets to be taken with the same schedule as the active treatment.
Trial information was received from ClinicalTrials.gov and was last updated in July 2016.
Information provided to ClinicalTrials.gov by Vertex Pharmaceuticals Incorporated.