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A Phase 3 Study of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor

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ClinicalTrials.gov Identifier: NCT02412111
Recruitment Status : Completed
First Posted : April 8, 2015
Last Update Posted : October 23, 2017
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group, multicenter study in subjects aged 12 years and older with CF who are heterozygous for the F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive.

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 156 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Ivacaftor-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, Heterozygous for the F508del-CFTR Mutation and a Second CFTR Allele With a Gating Defect That Is Clinically Demonstrated to be Ivacaftor Responsive
Study Start Date : June 2015
Actual Primary Completion Date : September 2017
Actual Study Completion Date : September 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arm Intervention/treatment
Active Comparator: VX-661/ ivacaftor

AM: VX-661 100-mg/ivacaftor 150-mg fixed-dose tablet ivacaftor matching placebo tablet oral

PM: ivacaftor 150-mg tablet oral

Drug: ivacaftor
Other Name: VX-770

Drug: VX-661
Drug: Placebo
Active Comparator: ivacaftor

AM: VX-661/ivacaftor matching placebo tablet ivacaftor 150-mg tablet oral

PM: ivacaftor 150-mg tablet oral

Drug: ivacaftor
Other Name: VX-770

Drug: Placebo



Primary Outcome Measures :
  1. Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: from baseline through Week 8 ]

Secondary Outcome Measures :
  1. Relative change in percent predicted FEV1 [ Time Frame: from baseline through Week 8 ]
  2. Absolute change in sweat chloride [ Time Frame: from baseline through Week 8 ]
  3. Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline through Week 8 ]
  4. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Screening through 4 weeks after receiving last dose ]
  5. PK parameters of VX-661, M1-661, ivacaftor, and M1-ivacaftor including area under the plasma concentration versus time curve (AUC) and peak plasma concentration (Cmax) as determined by population PK analysis [ Time Frame: From Week -2 through Week 2 ]


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

Inclusion Criteria:

  • Heterozygous for F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive
  • FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height during screening
  • 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.
  • Pregnant and nursing females (females of childbearing potential must have a negative pregnancy test at Screening and Week -4 Visits).
  • 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): NCT02412111


  Show 68 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02412111     History of Changes
Other Study ID Numbers: VX14-661-109
First Posted: April 8, 2015    Key Record Dates
Last Update Posted: October 23, 2017
Last Verified: August 2017

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