Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation
NCT ID: NCT01531673
Last Updated: 2018-04-13
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
194 participants
INTERVENTIONAL
2012-02-29
2014-03-31
Brief Summary
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Detailed Description
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This study is separated into seven groups: Group 1-7, respectively. Approximately 180 participants were randomized in a ratio of 4:1; active drug to matching placebo in each group.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Group 1-6d Combined: Placebo
All participants in group 1, 2a, 2b, 3a, 3b, 4, 5a, 5b, 6a and 6d who received placebo matched to VX-661 tablet and/or placebo matched to ivacaftor tablet for up to 28 days.
Placebo matched to VX-661
Placebo matched to ivacaftor
Group 1: VX-661 10 mg qd
All participants in group 1 who received VX-661 10 milligram (mg) tablet orally once daily (qd) for up to 28 days.
VX-661
Group 2a: VX-661 30 mg qd
All participants in group 2a who received VX-661 30 mg tablet orally qd and placebo matched to Ivacaftor tablet every 12 hours (q12h) for up to 28 days.
VX-661
Placebo matched to ivacaftor
Group 2b: VX-661 10 mg qd/Ivacaftor 150 mg q12h
All participants in group 2b who received VX-661 10 mg tablet qd and Ivacaftor 150 mg tablet q12h orally for up to 28 days.
VX-661
Ivacaftor
Group 3a: VX-661 100 mg qd
All participants in group 3a who received VX-661 100 mg tablet orally qd and placebo matched to Ivacaftor tablet q12h for up to 28 days.
VX-661
Placebo matched to ivacaftor
Group 3b: VX-661 30 mg qd/Ivacaftor 150 mg q12h
All participants in group 3b who received VX-661 30 mg tablet qd and Ivacaftor 150 mg tablet q12h orally for up to 28 days.
VX-661
Ivacaftor
Group 4: VX-661 100 mg qd/Ivacaftor 150 mg q12h
All participants in group 4 who received VX-661 100 mg tablet qd and Ivacaftor 150 mg tablet q12h orally for up to 28 days.
VX-661
Ivacaftor
Group 5a: VX-661 150 mg qd
All participants in group 5a who received VX-661 150 mg tablet orally qd for up to 28 days.
VX-661
Group 5b: VX-661 150 mg qd/Ivacaftor 150 mg q12h
All participants in group 5b who received VX-661 150 mg tablet qd and Ivacaftor 150 mg tablet q12h orally for up to 28 days.
VX-661
Ivacaftor
Group 6a: VX-661 100 mg qd/Ivacaftor 50 mg q12h
All participants in group 6a who received VX-661 100 mg tablet qd and Ivacaftor 50 mg tablet q12h orally for up to 28 days.
VX-661
Ivacaftor
Group 6d: VX-661 50 mg q12h/Ivacaftor 150 mg q12h
All participants in group 6d who received VX-661 50 mg tablet and Ivacaftor 150 mg tablet q12h orally for up to 28 days.
VX-661
Ivacaftor
Group 7: Placebo
All participants in group 7 who received placebo matched to VX-661 tablet orally qd in combination with physician-prescribed Kalydeco (Ivacaftor) for up to 28 days.
Ivacaftor
Placebo matched to VX-661
Group 7: VX-661 100 mg qd
All participants in group 7 who received VX-661 100 mg tablet orally qd in combination with physician-prescribed Kalydeco (Ivacaftor) for up to 28 days.
VX-661
Ivacaftor
Interventions
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VX-661
Ivacaftor
Placebo matched to VX-661
Placebo matched to ivacaftor
Eligibility Criteria
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Inclusion Criteria
* Must have the F508del-CFTR gene mutation in both alleles (Groups 1, 2, 3, 4, 5, 6). Group 7 participants must have the F508del-CFTR mutation on 1 allele, and gating mutation G551D on the second allele and have been on their physician prescribed 150 mg KalydecoTM q12h (commercially available ivacaftor) for at least 28 days at the Screening Visit.
* Forced expiratory volume in 1 second(FEV1) 40% to 90% (inclusive) of predicted normal for age, gender, and height (Knudson standards) at screening
* Weight \>40 kg and BMI \>18.5
* Participants of child-bearing potential and who are sexually active must meet the contraception requirements.
Exclusion Criteria
* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Study Day 1.
* History of solid organ or hematological transplantation
* Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives (whichever is longer) before screening
* History of alcohol, medication, or illicit drug abuse within 1 year prior to screening
* Pregnant, breast-feeding, or not willing to follow contraception requirements
12 Years
ALL
No
Sponsors
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Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Principal Investigators
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Scott Donaldson, MD
Role: PRINCIPAL_INVESTIGATOR
University of North Carolina
Locations
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Vertex Investigational Site
Birmingham, Alabama, United States
Vertex Investigational Site
Oakland, California, United States
Vertex Investigational Site
Boise, Idaho, United States
Vertex Investigational Site
Chicago, Illinois, United States
Vertex Investigational Site
Boston, Massachusetts, United States
Vertex Investigational Site
Grand Rapids, Michigan, United States
Vertex Investigational Site
Kansas City, Missouri, United States
Vertex Investigational Site
Long Branch, New Jersey, United States
Vertex Investigational Site
New Hyde Park, New York, United States
Vertex Investigational Site
Chapel Hill, North Carolina, United States
Vertex Investigational Site
Cincinnati, Ohio, United States
Vertex Investigational Site
Columbus, Ohio, United States
Vertex Investigational Site
Oklahoma City, Oklahoma, United States
Vertex Investigational Site
Hershey, Pennsylvania, United States
Vertex Investigational Site
Pittsburgh, Pennsylvania, United States
Vertex Investigational Site
Charleston, South Carolina, United States
Vertex Investigational Site
Salt Lake City, Utah, United States
Vertex Investigational Site
Burlington, Vermont, United States
Vertex Investigational Site
Seattle, Washington, United States
Vertex Investigational Site
Calgary, Alberta, Canada
Vertex Investigational Site
Vancouver, British Columbia, Canada
Vertex Investigational Site
Halifax, Nova Scotia, Canada
Vertex Investigational Site
Toronto, Ontario, Canada
Vertex Investigational Site
Erlangen, Bavaria, Germany
Vertex Investigational Site
Frankfurt am Main, Hesse, Germany
Vertex Investigational Site
Hanover, Lower Saxony, Germany
Vertex Investigational Site
Cologne, North Rhine-Westphalia, Germany
Vertex Investigational Site
Berlin, , Germany
Vertex Investigational Site
Bochum, , Germany
Vertex Investigational Site
Jena, , Germany
Vertex Investigational Site
Munich, , Germany
Vertex Investigational Site
Cambridge, Cambridgeshire, United Kingdom
Vertex Investigational Site
London, Greater London, United Kingdom
Vertex Investigational Site
Manchester, Greater Manchester, United Kingdom
Vertex Investigational Site
Southhampton, Hampshire, United Kingdom
Vertex Investigational Site
Cardiff, Vale of Glamorgen, United Kingdom
Countries
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Other Identifiers
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2011-003821-93
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX11-661-101
Identifier Type: -
Identifier Source: org_study_id
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