A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation
NCT ID: NCT02514473
Last Updated: 2017-10-23
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
206 participants
INTERVENTIONAL
2015-07-31
2016-09-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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LUM/IVA
Fixed-dose combination with lumacaftor (LUM) 200 mg every 12 hours (q12h)/ ivacaftor (IVA) 250 mg q12h
VX-809
VX-770
Placebo
Matching placebo q12h
Placebo
Interventions
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VX-809
Placebo
VX-770
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Subjects with confirmed diagnosis of CF at the Screening Visit.
* Subjects who are homozygous for the F508del CFTR mutation
* Subjects with ppFEV1 of ≥70 percentage points adjusted for age, sex, and height
* Subjects with a screening LCI2.5 result greater than or equal to 7.5
Exclusion Criteria
* Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject
* Clinically significant abnormalities in hemoglobin, liver function, or renal function at the Screening Visit.
* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
* History of solid organ or hematological transplantation at the Screening Visit
6 Years
11 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
Los Angeles, California, United States
Palo Alto, California, United States
Aurora, Colorado, United States
Wilmington, Delaware, United States
Orlando, Florida, United States
Atlanta, Georgia, United States
Chicago, Illinois, United States
Iowa City, Iowa, United States
Boston, Massachusetts, United States
Minneapolis, Minnesota, United States
Kansas City, Missouri, United States
Omaha, Nebraska, United States
Manchester, New Hampshire, United States
Chapel Hill, North Carolina, United States
Cincinnati, Ohio, United States
Cleveland, Ohio, United States
Dayton, Ohio, United States
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
Charleston, South Carolina, 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
Herston, , Australia
New South Wales, , Australia
Parkville, , Australia
Subiaco, , Australia
Westmead, , Australia
Brussels, , Belgium
Leuven, , Belgium
Vancouver, British Columbia, Canada
Toronto, Ontario, Canada
Montreal, Quebec, Canada
Copenhagen, , Denmark
Bordeaux, , France
Bron, , France
Paris, , France
Berlin, , Germany
Cologne, , Germany
Giessen, , Germany
Hanover, , Germany
Munich, , Germany
Stockholm, , Sweden
Edinburgh, Lothian Region, United Kingdom
Leeds, West Yorkshire, United Kingdom
Belfast, , United Kingdom
London, , United Kingdom
Countries
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References
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Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Ratjen F, Hug C, Marigowda G, Tian S, Huang X, Stanojevic S, Milla CE, Robinson PD, Waltz D, Davies JC; VX14-809-109 investigator group. Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial. Lancet Respir Med. 2017 Jul;5(7):557-567. doi: 10.1016/S2213-2600(17)30215-1. Epub 2017 Jun 9.
Other Identifiers
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VX14-809-109
Identifier Type: -
Identifier Source: org_study_id