Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation
ENVISION
A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Pharmacokinetics, Efficacy and Safety of VX-770 in Subjects Aged 6 to 11 Years With Cystic Fibrosis and the G551D Mutation
1 other identifier
interventional
52
7 countries
29
Brief Summary
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 6 to 11 years who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic adenosine monophosphate (AMP)-dependent protein kinase A (PKA) activation.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Aug 2009
29 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 26, 2009
CompletedFirst Posted
Study publicly available on registry
May 28, 2009
CompletedStudy Start
First participant enrolled
August 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2010
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2011
CompletedResults Posted
Study results publicly available
August 21, 2012
CompletedAugust 21, 2012
July 1, 2012
1.3 years
May 26, 2009
February 27, 2012
July 18, 2012
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24
Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
baseline through 24 weeks
Secondary Outcomes (4)
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 48
baseline through 48 weeks
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Through Week 24 and Week 48 (Respiratory Domain Score, Children)
baseline through 24 weeks and 48 weeks
Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48
baseline through 24 weeks and 48 weeks
Absolute Change From Baseline in Weight at Week 24 and Week 48
baseline to 24 weeks and 48 weeks
Study Arms (2)
Placebo
PLACEBO COMPARATORSubjects who received placebo every 12 hours (q12h) for up to 48 weeks.
150 mg Ivacaftor q12h
EXPERIMENTALSubjects who received 150 mg of ivacaftor q12h for up to 48 weeks.
Interventions
Eligibility Criteria
You may qualify if:
- Weighing at least 15 kg
- 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
You may not qualify if:
- History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
- 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)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Vertex Pharmaceuticals Incorporatedlead
- Cystic Fibrosis Foundationcollaborator
Study Sites (29)
University of Alabama
Birmingham, Alabama, 35233-1711, United States
Emory Cystic Fibrosis Center
Atlanta, Georgia, 30322, United States
Children's Memorial Hospital
Chicago, Illinois, 60614, United States
The Cystic Fibrosis Center of Chicago
Glenview, Illinois, 60025, United States
Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
University of Iowa Department of Pediatrics
Iowa City, Iowa, 52242, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Children's Hospital Boston
Boston, Massachusetts, 02115, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
Helen DeVos Children's Hospital Spectrum Health Hospitals
Grand Rapids, Michigan, 49503, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
The Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
University of Nebraska Medical Center Pediatric Pulmonary/ CF
Omaha, Nebraska, 68195-5190, United States
East Tennessee Children's Hospital Pediatric Pulmonary and Respiratory Care
Knoxville, Tennessee, 37916, United States
University of Utah Pediatric Pulmonology
Salt Lake City, Utah, 84108, United States
University of Virginia Pediatric Respiratory Medicine
Charlottesville, Virginia, 22908, United States
The Children's Hospital Westmead
Westmead, New South Wales, 2145, Australia
Royal Children's Hospital Brisbane
Herston, Queensland, 4029, Australia
Royal Children's Hospital Melbourne
Parkville, Victoria, 3052, Australia
Princess Margaret Hospital for Children
Subiaco, Western Australia, 6008, Australia
British Columbia Children's Hospital
Vancouver, British Columbia, V6H-3V4, Canada
Hospital for Sick Children CF Center
Toronto, Ontario, M5G 1X8, Canada
Hôpital Robert Debré - Service de gastro-entérologiemucoviscidose et nutrition
Paris, 75935, France
Kinder- und Jugendklinik Universitätsklinikum Erlangen
Erlangen, 91054, Germany
Mukoviszidose-Zentrum am Klinikum der Friedrich-Schiller-Universität Jena, Klinik für Kinder- und Jugendmedizin
Jena, 07740, Germany
Our Lady's Children's Hospital
Dublin, 12, Ireland
The National Children's Hospital
Dublin, 24, Ireland
Dept of Gene Therapy, Imperial College London
London, SW3 6LR, United Kingdom
Related Publications (1)
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.
PMID: 23590265DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex
Study Officials
- PRINCIPAL INVESTIGATOR
Richard Ahrens, MD
Roy A. & Lucille A. Carver College of Medicine
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 26, 2009
First Posted
May 28, 2009
Study Start
August 1, 2009
Primary Completion
November 1, 2010
Study Completion
April 1, 2011
Last Updated
August 21, 2012
Results First Posted
August 21, 2012
Record last verified: 2012-07