Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
NCT ID: NCT01225211
Last Updated: 2015-10-05
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
312 participants
INTERVENTIONAL
2010-10-31
2014-04-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
FACTORIAL
TREATMENT
TRIPLE
Study Groups
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Cohort 1: Placebo
Participants homozygous (HO) for the F508del-CF transmembrane conductance regulator gene (CFTR) mutation received lumacaftor matched placebo once daily (qd) (Day 1 through Day 14), followed by lumacaftor matched placebo qd in combination with ivacaftor matched placebo every 12 hours (q12h) (Day 15 through Day 21).
Lumacaftor Placebo
Matching placebo tablet.
Ivacaftor Placebo
Matching placebo tablet.
Cohort 1: LUM 200 mg qd/LUM 200 mg qd+IVA 150 mg q12h
Participants homozygous for the F508del-CFTR mutation received 200 milligram (mg) of lumacaftor (LUM) qd (Day 1 through Day 14), followed by 200 mg of lumacaftor qd in combination with 150 mg of ivacaftor (IVA) q12h (Day 15 through Day 21).
Lumacaftor
Tablet
Ivacaftor
Tablet.
Cohort 1: LUM 200 mg qd/LUM 200 mg qd+IVA 250 mg q12h
Participants homozygous for the F508del-CFTR mutation received 200 mg of lumacaftor alone qd (Day 1 through Day 14), followed by 200 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 15 through Day 21).
Lumacaftor
Tablet
Ivacaftor
Tablet.
Cohort 2 and 3: Placebo (HO and HE)
Participants homozygous or heterozygous for the F508del-CFTR mutation received lumacaftor matched placebo qd (Day 1 through Day 28), followed by lumacaftor matched placebo in combination with ivacaftor matched placebo q12h (Day 29 through Day 56).
Lumacaftor Placebo
Matching placebo tablet.
Ivacaftor Placebo
Matching placebo tablet.
Cohort 2: LUM 200 mg qd/LUM 200 mg qd+IVA 250 mg q12h (HO)
Participants homozygous for the F508del-CFTR mutation received 200 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 200 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Lumacaftor
Tablet
Ivacaftor
Tablet.
Cohort 2: LUM 400 mg qd/LUM 400 mg qd+IVA 250 mg q12h (HO)
Participants homozygous for the F508del-CFTR mutation received 400 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 400 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Lumacaftor
Tablet
Ivacaftor
Tablet.
Cohort 2: LUM 600 mg qd/LUM 600 mg qd+IVA 250 mg q12h (HO&HE)
Participants homozygous or heterozygous for the F508del-CFTR mutation received 600 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 600 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Lumacaftor
Tablet
Ivacaftor
Tablet.
Cohort 3: LUM 400 mg q12h/LUM 400 mg q12h+IVA 250 mg q12h (HO)
Participants homozygous for the F508del-CFTR mutation received 400 mg of lumacaftor alone q12h (Day 1 through Day 28), followed by 400 mg of lumacaftor q12h in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56).
Lumacaftor
Tablet
Ivacaftor
Tablet.
Cohort 4: Placebo
Participants heterozygous for the F508del-CFTR mutation received lumacaftor matched placebo in combination with ivacaftor matched placebo q12h (Day 1 through Day 56).
Lumacaftor Placebo
Matching placebo tablet.
Ivacaftor Placebo
Matching placebo tablet.
Cohort 4: LUM 400 mg q12h+IVA 250 mg q12h
Participants heterozygous for the F508del-CFTR mutation received 400 mg of lumacaftor q12h in combination with 250 mg of ivacaftor q12h (Day 1 through Day 56).
Lumacaftor
Tablet
Ivacaftor
Tablet.
Interventions
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Lumacaftor
Tablet
Ivacaftor
Tablet.
Lumacaftor Placebo
Matching placebo tablet.
Ivacaftor Placebo
Matching placebo tablet.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Must have the F508del-CFTR mutation on at least 1 allele.
* FEV1 greater than equal (\>=) 40% of predicted normal for age, gender, and height (Knudson standards)(Cohort 1, 2, and 3); FEV1 40-90% of predicted normal for age, gender, and height (Hankinson standards (Cohort 4)
* Participant of child-bearing potential and who are sexually active must meet the contraception requirements
Exclusion Criteria
* An acute illness including acute upper or lower respiratory infection, pulmonary exacerbation or changes in therapy (including antibiotics) for pulmonary disease within 14 days (Cohort 1, 2, and 3) or 28 days (Cohort 4) before receiving the first dose of study drug.
* History of solid organ or hematological transplantation.
* History of alcohol abuse or drug addiction in the past year, including cannabis, cocaine, and opiates.
* Ongoing participation in another therapeutic clinical study, or prior participation in an investigational drug study without appropriate washout
* Pregnant or nursing females; females of childbearing potential who are unwilling or unable to use an acceptable method of non-hormonal contraception
* Participants enrolled in Cohort 1 or Cohort 2 will not be eligible for Cohort 3
* Ongoing or prior participation in an investigational drug study within 30 days of the Screening Visit
* Heterozygous participants who participated in Cohort 2 and meet the eligibility criteria for Cohort 4 may participate in Cohort 4
* Evidence of lens opacity or cataract as determined by the ophthalmologic examination (Cohort 4 only)
18 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
La Jolla, California, United States
Long Beach, California, United States
Palo Alto, California, United States
Sacramento, California, United States
Denver, Colorado, United States
New Haven, Connecticut, United States
Miami, Florida, United States
Orlando, Florida, United States
Tampa, Florida, United States
Atlanta, Georgia, United States
Boise, Idaho, United States
Chicago, Illinois, United States
Iowa City, Iowa, United States
Kansas City, Kansas, United States
Lexington, Kentucky, United States
New Orleans, Louisiana, United States
Baltimore, Maryland, United States
Boston, Massachusetts, United States
Minneapolis, Minnesota, United States
St Louis, Missouri, United States
Omaha, Nebraska, United States
Lebanon, New Hampshire, United States
Buffalo, New York, United States
New Hyde Park, New York, United States
New York, New York, United States
Rochester, New York, United States
Syracuse, New York, United States
Chapel Hill, North Carolina, United States
Akron, Ohio, United States
Cleveland, Ohio, United States
Columbus, 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
Adelaide, , Australia
Brisbane, , Australia
Chermside, , Australia
Nedlands, , Australia
Parkville Victoria, , Australia
Westmead, , Australia
Leuven, , Belgium
Pierre-Bénite, Rhone, France
Paris, , France
Jena, Thuringia, Germany
Berlin, , Germany
Bochum, , Germany
Cologne, , Germany
Essen, , Germany
Auckland, , New Zealand
Christchurch, , New Zealand
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.
Rowe SM, McColley SA, Rietschel E, Li X, Bell SC, Konstan MW, Marigowda G, Waltz D, Boyle MP; VX09-809-102 Study Group. Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR. Ann Am Thorac Soc. 2017 Feb;14(2):213-219. doi: 10.1513/AnnalsATS.201609-689OC.
Boyle MP, Bell SC, Konstan MW, McColley SA, Rowe SM, Rietschel E, Huang X, Waltz D, Patel NR, Rodman D; VX09-809-102 study group. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med. 2014 Jul;2(7):527-38. doi: 10.1016/S2213-2600(14)70132-8. Epub 2014 Jun 24.
Other Identifiers
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2010-020413-90
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX09-809-102
Identifier Type: -
Identifier Source: org_study_id
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