Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
NCT ID: NCT02544451
Last Updated: 2021-05-24
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
246 participants
INTERVENTIONAL
2015-08-31
2020-04-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Treatment Period 1: LUM/IVA to LUM/IVA
LUM/IVA
Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).
LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).
Treatment Period 1: Placebo (PBO) to LUM/IVA
LUM/IVA
Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).
LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).
Treatment Period 1: Observational Cohort
No interventions assigned to this group
Treatment Period 2: LUM/IVA
LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Interventions
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LUM/IVA
Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).
LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).
LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not permanently discontinue treatment
* Willing to remain on a stable CF medication through the Safety Follow-up Visit.
Subjects entering the Observational Cohort must meet 1 of the following criteria:
* Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B.
* Received at least 4 weeks of study drug and completed visits up to Week 24 of Study 109 or Week 26 of Study 011B.
Exclusion Criteria
* Pregnant and nursing females.
* Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
* History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator
* History of poor compliance with study drug and/or procedure in the previous study as deemed by the investigator.
* Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor).
6 Years
ALL
No
Sponsors
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Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Locations
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Birmingham, Alabama, United States
Tucson, Arizona, United States
Long Beach, California, United States
Palo Alto, California, United States
Aurora, Colorado, United States
Wilmington, Delaware, United States
Jacksonville, Florida, United States
Orlando, Florida, United States
Atlanta, Georgia, United States
Chicago, Illinois, United States
Indianapolis, Indiana, United States
Iowa City, Iowa, United States
Boston, Massachusetts, United States
Minneapolis, Minnesota, United States
Kansas City, Missouri, United States
St Louis, Missouri, United States
Omaha, Nebraska, United States
Lebanon, New Hampshire, United States
Buffalo, New York, United States
Syracuse, New York, United States
Chapel Hill, North Carolina, United States
Cincinnati, Ohio, United States
Cleveland, Ohio, United States
Dayton, Ohio, United States
Portland, Oregon, United States
Pittsburgh, Pennsylvania, United States
Charleston, South Carolina, United States
Austin, Texas, United States
Houston, Texas, United States
Salt Lake City, Utah, United States
Colchester, Vermont, United States
Charlottesville, Virginia, United States
Norfolk, Virginia, United States
Richmond, Virginia, United States
Seattle, Washington, United States
Madison, Wisconsin, United States
Milwaukee, Wisconsin, United States
Parkville, Victoria, Australia
Herston, , Australia
New Lambton Heights, , Australia
Subiaco, , Australia
Westmead, , Australia
Brussels, , Belgium
Leuven, , Belgium
Vancouver, British Columbia, Canada
Toronto, Ontario, Canada
Montreal, Quebec, Canada
Copenhagen, , Denmark
Bron, Cedex, France
Bordeaux, , France
Paris, , France
Berlin, , Germany
Cologne, , Germany
Giessen, , Germany
Hanover, , Germany
Stockholm, , Sweden
Leeds, West Yorkshire, United Kingdom
Belfast, , United Kingdom
Edinburgh, , United Kingdom
London, , United Kingdom
Countries
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References
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Chilvers MA, Davies JC, Milla C, Tian S, Han Z, Cornell AG, Owen CA, Ratjen F. Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study. Lancet Respir Med. 2021 Jul;9(7):721-732. doi: 10.1016/S2213-2600(20)30517-8. Epub 2021 Jan 28.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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VX15-809-110
Identifier Type: -
Identifier Source: org_study_id
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