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

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ClinicalTrials.gov Identifier: NCT02347657
Recruitment Status : Completed
First Posted : January 27, 2015
Last Update Posted : February 16, 2018
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief 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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-661/ ivacaftor Drug: ivacaftor Drug: VX-661/ ivacaftor placebo Drug: ivacaftor placebo Phase 3

Detailed 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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 509 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
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
Study Start Date : January 2015
Actual Primary Completion Date : January 20, 2017
Actual Study Completion Date : January 20, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor
U.S. FDA Resources

Arm Intervention/treatment
Experimental: VX-661/ ivacaftor
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)
Drug: VX-661/ ivacaftor Drug: ivacaftor
Placebo Comparator: Placebo
0-mg film coated matching placebo tablets for oral administration in morning and evening
Drug: VX-661/ ivacaftor placebo Drug: ivacaftor placebo



Primary Outcome Measures :
  1. Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: baseline through Week 24 ]

Secondary Outcome Measures :
  1. Relative change in percent predicted FEV1 [ Time Frame: baseline through Week 24 ]
  2. Number of pulmonary exacerbations [ Time Frame: through Week 24 ]
  3. Absolute change in body mass index (BMI) [ Time Frame: baseline at Week 24 ]
  4. Absolute change in Cystic Fibrosis Questionnaire - Revised (CFQ-R) respiratory domain score [ Time Frame: baseline through Week 24 ]
  5. 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 ]
  6. Time-to-first pulmonary exacerbation [ Time Frame: through Week 24 ]
  7. Absolute change in sweat chloride [ Time Frame: baseline through Week 24 ]
  8. Absolute change in BMI z-score (in subjects <20 years of age at time of screening) [ Time Frame: baseline at Week 24 ]
  9. Absolute change in body weight [ Time Frame: baseline at Week 24 ]
  10. 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 ]
  11. 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 ]


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02347657


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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02347657     History of Changes
Other Study ID Numbers: VX14-661-106
First Posted: January 27, 2015    Key Record Dates
Last Update Posted: February 16, 2018
Last Verified: February 2017

Keywords provided by Vertex Pharmaceuticals Incorporated:
Homozygous for the F508del CFTR Mutation

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action