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 March 2015
End date March 2017
Trial size 300 participants
Trial identifier NCT02392234, 2014-004788-18, VX14-661-108

Summary

The purpose of this study is to evaluate the efficacy of VX-661 in combination with ivacaftor and ivacaftor monotherapy in subjects with Cystic Fibrosis (CF).

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model crossover assignment
Masking double blind (subject, caregiver, investigator)
Primary purpose treatment
Arm
(Experimental)
Morning dose: 1 tablet fixed-dose combination of VX-661 100 mg/ivacaftor 150 mg and 1 tablet ivacaftor placebo. Evening dose: 1 tablet ivacaftor 150 mg
vx-661/ivacaftor
ivacaftor
ivacaftor placebo
(Experimental)
Morning dose: 1 tablet placebo visually matched to the fixed-dose combination tablet and 1 tablet ivacaftor 150 mg Evening dose: 1 tablet ivacaftor 150 mg
ivacaftor
vx-661/ ivacaftor placebo
(Placebo Comparator)
Morning dose: 1 tablet placebo visually matched to the fixed-dose combination tablet and 1 tablet placebo visually matched to ivacaftor 150 mg Evening dose: 1 tablet placebo visually matched to ivacaftor 150 mg
vx-661/ ivacaftor placebo
ivacaftor placebo

Primary Outcomes

Measure
Absolute Change From Study Baseline to Average of Week 4 and Week 8 Measurements in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)
time frame: Baseline, Week 4 and Week 8 of each treatment period

Secondary Outcomes

Measure
Absolute Change From Study Baseline to Average of Week 4 and Week 8 Measurements in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
time frame: Baseline, Week 4 and Week 8 of each treatment period
Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values, standard digital electrocardiograms (ECGs), vital signs, pulse oximetry, and spirometry
time frame: Baseline up to 28 days after last dose (up to 28 weeks)
Relative Change From Study Baseline to Average of Week 4 and Week 8 Measurements in Percent Predicted FEV1
time frame: Baseline, Week 4 and Week 8 of each treatment period
Absolute Change From Study Baseline to Average of Week 4 and Week 8 Measurements in Sweat Chloride
time frame: Baseline, Week 4 and Week 8 of each treatment period
Pharmacokinetic parameters; Area under the plasma concentration versus time curve (AUC), Peak Plasma Concentration (Cmax) of VX-661, M1-661, 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: - Heterozygous for F508del-CFTR and a second allele with a CFTR mutation predicted to have residual function - Forced Expiratory Volume in 1 Second (FEV1) greater than or equal to (≥) 40 percent (%) and less than or equal to (≤) 90% of predicted normal for age, sex, and height during screening. - Sweat chloride value ≥60 millimole per liter (mmol/L) from test results obtained during screening OR as documented in the subject's medical record. - Sweat chloride value less than (<) 60 mmol/L must have documented evidence of chronic sinopulmonary disease - Stable CF disease as judged by the investigator 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 - A 12-lead electrocardiogram (ECG) demonstrating corrected QT interval (QTc) greater than (>) 450 milliseconds (msec) at the Screening Visit - History of solid organ or hematological transplantation - History or evidence of cataract, lens opacity, Y-suture, or lamellar rings determined to be clinically significant by the ophthalmologist during the ophthalmologic examination during the Screening Period - Ongoing or prior participation in an investigational drug study (including studies investigating VX-661, lumacaftor [VX-809], and/or ivacaftor) within 30 days of screening - Use of restricted medications or foods within the specified window before the first dose of study drug - Pregnant and 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 - Colonization with organisms associated with a more rapid decline in pulmonary status

Additional Information

Official title A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation, and a Second Allele With a CFTR Mutation Predicted to Have Residual Function
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Vertex Pharmaceuticals Incorporated.