A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
NCT ID: NCT01807949
Last Updated: 2016-09-27
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
563 participants
INTERVENTIONAL
2013-04-30
2014-04-30
Brief Summary
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Detailed Description
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The study included a Screening Period (Day -28 through Day -1), a Treatment Period (Day 1 \[first dose of study drug\] to Week 24 ± 5 days), and a Safety Follow-up Visit (4 weeks ± 7 days after the Week 24 Visit).
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Placebo
Placebo matched to lumacaftor (LUM, VX-809) and ivacaftor (IVA, VX-770) tablet every 12 hours (q12h), up to Week 24.
Placebo
Matching placebo tablet
LUM 600 mg qd/IVA 250 mg q12h
LUM 600 milligram (mg) plus IVA 250 mg fixed-dose combination (FDC) tablet in the morning and IVA 250 mg film-coated tablet in the evening, up to Week 24.
Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet
Ivacaftor
Film-coated tablet
LUM 400 mg q12h/ IVA 250 mg q12h
LUM 400 mg plus IVA 250 mg FDC tablet in the morning and in the evening, up to Week 24.
Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet
Interventions
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Placebo
Matching placebo tablet
Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet
Ivacaftor
Film-coated tablet
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Homozygous for the F508del CFTR mutation
* Forced expiratory volume in 1 second (FEV1) greater than or equal to (\>=) 40 percent (%) and less than or equal to (=\<) 90% of predicted normal for age, sex, and height
* Willing to remain on a stable CF medication regimen through Week 24 or, if applicable, the Safety Follow up Visit
Exclusion Criteria
* History of solid organ or hematological transplantation
* History of alcohol or drug abuse in the past year
* Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of screening.
* Use of strong inhibitors, moderate inducers, or strong inducers of Cytochrome P450 3A (CYP3A) within 14 days before Day 1 of dosing
12 Years
65 Years
ALL
No
Sponsors
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Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Locations
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Oakland, California, United States
Sacramento, California, United States
Aurora, Colorado, United States
Hartford, Connecticut, United States
New Haven, Connecticut, United States
Jacksonville, Florida, United States
Miami, Florida, United States
Indianapolis, Indiana, United States
Iowa City, Iowa, United States
Kansas City, Kansas, United States
Lexington, Kentucky, United States
Portland, Maine, United States
Worcester, Massachusetts, United States
Detroit, Michigan, United States
Grand Rapids, Michigan, United States
Minneapolis, Minnesota, United States
Jackson, Mississippi, United States
St Louis, Missouri, United States
Omaha, Nebraska, United States
Morristown, New Jersey, United States
New Brunswick, New Jersey, United States
Albuquerque, New Mexico, United States
Albany, New York, United States
Buffalo, New York, United States
New York, New York, United States
Rochester, New York, United States
Syracuse, New York, United States
Cleveland, Ohio, United States
Columbus, Ohio, United States
Toledo, Ohio, United States
Oklahoma City, Oklahoma, United States
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
Charleston, South Carolina, United States
Sioux Falls, South Dakota, United States
Memphis, Tennessee, United States
Nashville, Tennessee, United States
Dallas, Texas, United States
Fort Worth, Texas, United States
Houston, Texas, United States
San Antonio, Texas, United States
Salt Lake City, Utah, United States
Richmond, Virginia, United States
Seattle, Washington, United States
Spokane, Washington, United States
Milwaukee, Wisconsin, United States
Brisbane, Queensland, Australia
Chermside, Queensland, Australia
Herston, Queensland, Australia
South Brisbane, Queensland, Australia
Nedlands, Western Australia, Australia
Subiaco, Western Australia, Australia
Innsbruck, , Austria
Wels, , Austria
Brussels, , Belgium
Ghent, , Belgium
Leuven, , Belgium
Liège, , Belgium
Calgary, Alberta, Canada
Edmonton, Alberta, Canada
Vancouver, British Columbia, Canada
Montreal, Quebec, Canada
København Ø, , Denmark
Marseille, Bouches-du-Rhône, France
Toulouse, Haute Garonne, France
Montpellier, Herault, France
Lille, Nord, France
Paris, Paris, France
Bordeaux, , France
Munich, Bavaria, Germany
Frankfurt am Main, Hesse, Germany
Giessen, Hesse, Germany
Hanover, Niederachsen, Germany
Bochum, North Rhine-Westphalia, Germany
Jena, Thuringia, Germany
Barcelona, , Spain
Valencia, , Spain
Bristol, Avon, United Kingdom
London, Greater London, United Kingdom
Liverpool, Merseyside, United Kingdom
Newcastle upon Tyne, Tyne & Wear, United Kingdom
Leeds, West Yorkshire, 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.
Acaster S, Mukuria C, Rowen D, Brazier JE, Wainwright CE, Quon BS, Duckers J, Quittner AL, Lou Y, Sosnay PR, McGarry LJ. Development of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions: Estimating Utilities From the Cystic Fibrosis Questionnaire-Revised. Value Health. 2023 Apr;26(4):567-578. doi: 10.1016/j.jval.2022.12.002. Epub 2022 Dec 9.
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.
Flume PA, Suthoff ED, Kosinski M, Marigowda G, Quittner AL. Measuring recovery in health-related quality of life during and after pulmonary exacerbations in patients with cystic fibrosis. J Cyst Fibros. 2019 Sep;18(5):737-742. doi: 10.1016/j.jcf.2018.12.004. Epub 2018 Dec 23.
McColley SA, Konstan MW, Ramsey BW, Stuart Elborn J, Boyle MP, Wainwright CE, Waltz D, Vera-Llonch M, Marigowda G, Jiang JG, Rubin JL. Lumacaftor/Ivacaftor reduces pulmonary exacerbations in patients irrespective of initial changes in FEV1. J Cyst Fibros. 2019 Jan;18(1):94-101. doi: 10.1016/j.jcf.2018.07.011. Epub 2018 Aug 23.
Elborn JS, Ramsey BW, Boyle MP, Konstan MW, Huang X, Marigowda G, Waltz D, Wainwright CE; VX-809 TRAFFIC and TRANSPORT study groups. Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup: a pooled analysis. Lancet Respir Med. 2016 Aug;4(8):617-626. doi: 10.1016/S2213-2600(16)30121-7. Epub 2016 Jun 10.
Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Colombo C, Davies JC, De Boeck K, Flume PA, Konstan MW, McColley SA, McCoy K, McKone EF, Munck A, Ratjen F, Rowe SM, Waltz D, Boyle MP; TRAFFIC Study Group; TRANSPORT Study Group. Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. N Engl J Med. 2015 Jul 16;373(3):220-31. doi: 10.1056/NEJMoa1409547. Epub 2015 May 17.
Other Identifiers
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VX12-809-104
Identifier Type: -
Identifier Source: org_study_id
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