Safety Study of Ivacaftor in Subjects With Cystic Fibrosis
NCT ID: NCT00457821
Last Updated: 2012-10-05
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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COMPLETED
PHASE2
39 participants
INTERVENTIONAL
2007-05-31
2008-08-31
Brief Summary
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Detailed Description
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The study was conducted in 2 parts:
* Part 1 consisted of Group A and Group B. Subjects in Group A (10 subjects) were randomized to receive 25 mg of ivacaftor every 12 hours \[q12h\] (4 subjects), 75 mg of ivacaftor q12h (4 subjects), or placebo (2 subjects) for 14 days. Following a 7- to 28-day washout period, subjects who received active study drug crossed over to the alternate dose strength of ivacaftor for an additional 14 days. Placebo subjects continued to receive placebo for an additional 14 days. Subjects in Group B (10 subjects) were randomized to receive 75 mg of ivacaftor q12h (4 subjects), 150 mg of ivacaftor q12h (4 subjects), or placebo (2 subjects) for 14 days. Following a 7- to 28-day washout period, the subjects who received active study drug crossed over to the alternate dose strength of ivacaftor for an additional 14 days. Placebo subjects continued to receive placebo for an additional 14 days.
* Part 2 consisted of Group C; these subjects did not participate in Part 1. Subjects were randomized to receive 150 mg of ivacaftor q12h (7 subjects), 250 mg of ivacaftor q12h (7 subjects), or placebo (4 subjects) for a total of 28 days. Ivacaftor doses studied in Part 2 were selected following an interim pharmacokinetic/pharmacodynamic (PK/PD) and statistical analyses of data from Part 1. The 2 doses selected for Part 2 were anticipated to enable better definition of the optimal therapeutic dose.
Conditions
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Keywords
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Study Design
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RANDOMIZED
CROSSOVER
TREATMENT
QUADRUPLE
Study Groups
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Ivacaftor Group A
Subjects in Part 1 who first received 25 mg or 75 mg of ivacaftor every 12 hours (q12h) for 14 days, then crossed over to receive the alternate dose for another 14 days.
Ivacaftor 25 mg/75 mg
25 mg or 75 mg q12h for a total of 28 days (Part 1)
Ivacaftor Group B
Subjects in Part 1 who first received 75 mg or 150 mg of ivacaftor q12h for 14 days then crossed over to receive the alternate dose for another 14 days.
Ivacaftor 75 mg/150 mg
75 mg or 150 mg q12h for a total of 28 days (Part 1)
Ivacaftor Group C
Subjects in Part 2 who received 150 mg or 250 mg of ivacaftor q12h for 28 days.
Ivacaftor 150 mg or 250 mg
150 mg or 250 mg of ivacaftor q12h for 28 days (Part 2)
Placebo
Subjects who received placebo in Part 1 and subjects who received placebo in Part 2.
Placebo
Given q12h for 28 days each in Part 1 and Part 2 of the study
Interventions
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Ivacaftor 25 mg/75 mg
25 mg or 75 mg q12h for a total of 28 days (Part 1)
Ivacaftor 75 mg/150 mg
75 mg or 150 mg q12h for a total of 28 days (Part 1)
Ivacaftor 150 mg or 250 mg
150 mg or 250 mg of ivacaftor q12h for 28 days (Part 2)
Placebo
Given q12h for 28 days each in Part 1 and Part 2 of the study
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
* Forced expiratory volume in 1 second (FEV1) of at least 40% of predicted normal for age, gender, and height
* Willing to remain on stable medication regimen for the duration of study participation
* No significant clinical laboratory abnormalities, not pregnant, and willing to use at least 2 highly effective birth control methods during Part 1 and 1 highly effective birth control method during Part 2 of the study
* No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
Exclusion Criteria
* Ongoing acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 14 days of Day 1 of the study
* History of alcohol, medication or illicit drug abuse within one year prior to Day 1
* Abnormal liver function ≥ 3x the upper limit of normal
* History of abnormal renal function (creatinine clearance \< 50 mL/min using Cockcroft-Gault equation)
* History of solid organ or hematological transplantation
* Pregnant or breast-feeding (for women)
* Ongoing participation in another therapeutic clinical trial, or prior participation in an investigational drug study without appropriate washout
* Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP3A4)
18 Years
ALL
No
Sponsors
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Cystic Fibrosis Foundation
OTHER
Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Monitor
Role: STUDY_DIRECTOR
Vertex Pharmaceuticals Incorporated
Locations
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University of Alabama Hospital
Birmingham, Alabama, United States
Stanford University Medical Center
Palo Alto, California, United States
The Children's Hospital
Aurora, Colorado, United States
Roy J. and Lucille A. Carver College of Medicine, The University of Iowa
Iowa City, Iowa, United States
Johns Hopkins Hospital
Baltimore, Maryland, United States
Pulmonary and Critical Care Medicine, Massachusetts General Hospital
Boston, Massachusetts, United States
Children's Hospital of Boston
Boston, Massachusetts, United States
Division of Pulmonary, Allergy and Critical Care Medicine, University of Minnesota
Minneapolis, Minnesota, United States
Cystic Fibrosis Pulmonary Research and Treatment Center, University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Rainbow Babies and Children's Hospital
Cleveland, Ohio, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
Pulmonary Critical Care, University of Washington
Seattle, Washington, United States
Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children
Toronto, Ontario, Canada
CF Clinic, Pediatric Pulmonology and Neonatology, Medical School of Hannover
Straße, Hannover, Germany
Countries
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References
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Accurso FJ, Rowe SM, Clancy JP, Boyle MP, Dunitz JM, Durie PR, Sagel SD, Hornick DB, Konstan MW, Donaldson SH, Moss RB, Pilewski JM, Rubenstein RC, Uluer AZ, Aitken ML, Freedman SD, Rose LM, Mayer-Hamblett N, Dong Q, Zha J, Stone AJ, Olson ER, Ordonez CL, Campbell PW, Ashlock MA, Ramsey BW. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med. 2010 Nov 18;363(21):1991-2003. doi: 10.1056/NEJMoa0909825.
Accurso FJ, Van Goor F, Zha J, Stone AJ, Dong Q, Ordonez CL, Rowe SM, Clancy JP, Konstan MW, Hoch HE, Heltshe SL, Ramsey BW, Campbell PW, Ashlock MA. Sweat chloride as a biomarker of CFTR activity: proof of concept and ivacaftor clinical trial data. J Cyst Fibros. 2014 Mar;13(2):139-47. doi: 10.1016/j.jcf.2013.09.007.
Related Links
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Genetics Home Reference
Medline Plus
U.S. FDA Resources
Other Identifiers
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VX06-770-101
Identifier Type: -
Identifier Source: org_study_id