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: NCT01807923
Last Updated: 2015-08-31
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
559 participants
INTERVENTIONAL
2013-05-31
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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Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet
Ivacaftor
Film-coated tablet
Placebo
Matching placebo 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
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
Anchorage, Alaska, United States
Tucson, Arizona, United States
Little Rock, Arkansas, United States
Fresno, California, United States
La Jolla, California, United States
Loma Linda, California, United States
Long Beach, California, United States
Los Angeles, California, United States
Madera, California, United States
Oakland, California, United States
Palo Alto, California, United States
Denver, Colorado, United States
Gainsville, Florida, United States
Hollywood, Florida, United States
Orlando, Florida, United States
Tampa, Florida, United States
Atlanta, Georgia, United States
Boise, Idaho, United States
Chicago, Illinois, United States
Park Ridge, Illinois, United States
Peoria, Illinois, United States
Baltimore, Maryland, United States
Boston, Massachusetts, United States
Ann Arbor, Michigan, United States
Kansas City, Missouri, United States
Bedford, New Hampshire, United States
Lebanon, New Hampshire, United States
Long Branch, New Jersey, United States
Hawthorne, New York, United States
New York, New York, United States
Valhalla, New York, United States
Chapel Hill, North Carolina, United States
Durham, North Carolina, United States
Akron, Ohio, United States
Cincinnati, Ohio, United States
Dayton, Ohio, United States
Portland, Oregon, United States
Hershey, Pennsylvania, United States
Knoxville, Tennessee, United States
Austin, Texas, United States
Tyler, Texas, United States
Colchester, Vermont, United States
Charlottesville, Virginia, United States
Norfolk, Virginia, United States
Seattle, Washington, United States
Morgantown, West Virginia, United States
Madison, Wisconsin, United States
Broadmeadow, New South Wales, Australia
Westmead, New South Wales, Australia
Adelaide, South Australia, Australia
Halifax, British Columbia, Canada
Vancouver, British Columbia, Canada
Ottawa, Ontario, Canada
Toronto, Ontario, Canada
Brno, , Czechia
Plzen-Bory, , Czechia
Prague, , Czechia
Strasbourg, Bas Rhin, France
Roscoff, Finistere, France
Paris, Paris, France
Bron, Rhone, France
Pierre-Bénite, Rhone, France
Tübingen, Baden-Wurttemberg, Germany
Erlangen, Bavaria, Germany
Würzburg, Bavaria, Germany
Cologne, North Rhine-Westphalia, Germany
Leipzig, Saxony, Germany
Berlin, State of Berlin, Germany
Dublin, Dublin, Ireland
Tallaght, Dublin, Ireland
Florence, Firenze, Italy
Ancona, , Italy
Genova, , Italy
Milan, , Italy
Roma, , Italy
Verona, , Italy
Amsterdam, , Netherlands
Nijmegen, , Netherlands
Rotterdam, , Netherlands
The Hague, , Netherlands
Gothenburg, , Sweden
Stockholm, , Sweden
Uppsala, , Sweden
Belfast, Belfast, United Kingdom
Exeter, Devon, United Kingdom
Southhampton, Hampshire, United Kingdom
Nottingham, Nottinghamshire, United Kingdom
Birmingham, West Midlands, 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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VX12-809-103
Identifier Type: -
Identifier Source: org_study_id