Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation
NCT ID: NCT00909727
Last Updated: 2012-08-21
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
52 participants
INTERVENTIONAL
2009-08-31
2011-04-30
Brief Summary
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Detailed Description
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Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, ivacaftor was selected for clinical development as a possible treatment for patients with CF. Patients with the G551D mutation were the targeted population for this study because ivacaftor is a potentiator of the gating effect of the CFTR protein, and the most prevalent mutation with a gating defect in CF is the G551D mutation.
This study was conducted in 2 parts. Part A was conducted to analyze the PK properties of ivacaftor and to determine the most appropriate dose to administer to subjects in Part B of this study. Part B explored the safety and efficacy of ivacaftor over long-term treatment in subjects 6 to 11 years of age.
Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Placebo
Subjects who received placebo every 12 hours (q12h) for up to 48 weeks.
Placebo
Tablet given orally q12h for up to 48 weeks
150 mg Ivacaftor q12h
Subjects who received 150 mg of ivacaftor q12h for up to 48 weeks.
Ivacaftor
150-mg tablet given orally q12h for up to 48 weeks
Interventions
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Ivacaftor
150-mg tablet given orally q12h for up to 48 weeks
Placebo
Tablet given orally q12h for up to 48 weeks
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 40% to 105% (inclusive) of predicted normal for age, gender, and height (Knudson standards) at Screening
* Able to swallow tablets
* As judged by the investigator, parent or legal guardian and subject must have been able to understand protocol requirements, restrictions, and instructions, and the parent or legal guardian should have been able to ensure that the subject complied with, and was likely to complete, the study as planned
* Parent or legal guardian must have signed the informed consent form and corresponding assent must be obtained from the subject
* Willing to use at least 1 highly effective birth control method during the study
* No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
Exclusion Criteria
* Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 of the study
* Abnormal liver function ≥ 3x the upper limit of normal
* Abnormal renal function at Screening
* History of solid organ or hematological transplantation
* Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening
* Use of inhaled hypertonic saline treatment
* Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP 3A4)
6 Years
11 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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Richard Ahrens, MD
Role: PRINCIPAL_INVESTIGATOR
Roy A. & Lucille A. Carver College of Medicine
Locations
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University of Alabama
Birmingham, Alabama, United States
Emory Cystic Fibrosis Center
Atlanta, Georgia, United States
Children's Memorial Hospital
Chicago, Illinois, United States
The Cystic Fibrosis Center of Chicago
Glenview, Illinois, United States
Riley Hospital for Children
Indianapolis, Indiana, United States
University of Iowa Department of Pediatrics
Iowa City, Iowa, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Children's Hospital Boston
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Helen DeVos Children's Hospital Spectrum Health Hospitals
Grand Rapids, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
The Children's Mercy Hospital
Kansas City, Missouri, United States
University of Nebraska Medical Center Pediatric Pulmonary/ CF
Omaha, Nebraska, United States
East Tennessee Children's Hospital Pediatric Pulmonary and Respiratory Care
Knoxville, Tennessee, United States
University of Utah Pediatric Pulmonology
Salt Lake City, Utah, United States
University of Virginia Pediatric Respiratory Medicine
Charlottesville, Virginia, United States
The Children's Hospital Westmead
Westmead, New South Wales, Australia
Royal Children's Hospital Brisbane
Herston, Queensland, Australia
Royal Children's Hospital Melbourne
Parkville, Victoria, Australia
Princess Margaret Hospital for Children
Subiaco, Western Australia, Australia
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
Hospital for Sick Children CF Center
Toronto, Ontario, Canada
Hôpital Robert Debré - Service de gastro-entérologiemucoviscidose et nutrition
Paris, , France
Kinder- und Jugendklinik Universitätsklinikum Erlangen
Erlangen, , Germany
Mukoviszidose-Zentrum am Klinikum der Friedrich-Schiller-Universität Jena, Klinik für Kinder- und Jugendmedizin
Jena, , Germany
Our Lady's Children's Hospital
Dublin, , Ireland
The National Children's Hospital
Dublin, , Ireland
Dept of Gene Therapy, Imperial College London
London, , United Kingdom
Countries
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References
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Davies JC, Wainwright CE, Canny GJ, Chilvers MA, Howenstine MS, Munck A, Mainz JG, Rodriguez S, Li H, Yen K, Ordonez CL, Ahrens R; VX08-770-103 (ENVISION) Study Group. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. Am J Respir Crit Care Med. 2013 Jun 1;187(11):1219-25. doi: 10.1164/rccm.201301-0153OC.
Related Links
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Genetics Home Reference
Medline Plus
U.S. FDA Resources
Other Identifiers
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VX08-770-103
Identifier Type: -
Identifier Source: org_study_id