A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02347657
• Date of registration: 12/01/2015
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor
• Scientific 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
• Date of first enrolment: January 2015
• Target sample size: 510
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02347657
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 3

Countries of recruitment

Canada, Denmark, France, Germany, Ireland, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom, United States

Key inclusion & exclusion criteria

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

- Forced expiratory volume at one second (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
participant.

- 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 participants of reproductive potential who are not willing to follow
the contraception requirements



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: VX-661 Plus Ivacaftor Combination

Drug: Ivacaftor

Drug: VX-661 Plus Ivacaftor Combination Placebo

Drug: Ivacaftor placebo

Primary Outcome(s)

Absolute Change From Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 [Time Frame: Day 1, Through Week 24]

Secondary Outcome(s)

Absolute Change From Baseline (Day 1) in Body Weight at Week 24 [Time Frame: Day 1, Week 24]

Absolute Change From Baseline (Day 1) in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24 [Time Frame: Day 1, Through Week 24]

Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Day 1 up to Week 28]

Absolute Change From Baseline (Day 1) in Sweat Chloride Through Week 24 [Time Frame: Day 1, Through Week 24]

Number of Pulmonary Exacerbations Per Year [Time Frame: Day 1 through Week 24]

Absolute Change From Baseline (Day 1) Body Mass Index (BMI) at Week 24 [Time Frame: Day 1, Week 24]

Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661 and M2-VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA) [Time Frame: Pre-morning dose on Week 16]

Absolute Change From Baseline (Day 1) in BMI Z-score at Week 24 in Participants Less Than (<) 20 Years Old at the Time of Screening) [Time Frame: Day 1, Week 24]

Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24 [Time Frame: Day 1 through Week 24]

Relative Change From Baseline (Day 1) in ppFEV1 Through Week 24 [Time Frame: Day 1, Through Week 24]

Secondary ID(s)

VX14-661-106

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 12/06/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02347657