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Drug & research development » Clinical trials » NCT02347657

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

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

Clinical Trials gov number: NCT02347657

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Purpose: To evaluate the efficacy and safety of VX-661 in combination with ivacaftor in CF patients who are homozygous for the F508del CFTR mutation.

Phase: 3

Type: Interventional, randomized, double-blind, parallel assignment

Study sponsor: Vertex Pharmaceuticals Incorporated

  • 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

Intervention
Ivacaftor, VX-661

Geographical Location
United States

Number of Participants
101-1000 (≥12 years)

Primary Endpoint

Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) from baseline at Week 24

Secondary Endpoint

  • Relative change in percent predicted FEV1 from baseline to Week 24
  • Number of pulmonary exacerbations to Week 24
  • Absolute change in body mass index (BMI) from baseline to Week 24
  • Absolute change in Cystic Fibrosis Questionnaire – Revised (CFQ-R) respiratory domain score to 4 weeks after last dose from baseline to Week 24
  • Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values standard 12-lead electrocardiograms (ECGs), vital signs, and pulse oximetry to 4 weeks after last dose
  • Time-to-first pulmonary exacerbation
  • Absolute change in sweat chloride to Week 24
  • Absolute change in BMI z-score (in subjects <20 years of age at time of screening) from baseline at Week 24
  • Absolute change in body weight from baseline at Week 24
  • Pharmacokinetic parameters of VX-661, M1-661, M2-661, ivacaftor, and M1-ivacaftor, as determined by population analysis from Day 1 to Week 24
  • Pharmacokinetic parameters of VX-661, M1-661, M2-661, ivacaftor, and M1-ivacaftor, as determined by population analysis from Day 1 to Week 24

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  • 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)

  • Placebo Comparator:

0-mg film coated matching placebo tablets for oral administration in morning and evening

View Trial Results