Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation
STRIVE
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects With Cystic Fibrosis and the G551D Mutation
1 other identifier
interventional
167
8 countries
65
Brief Summary
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR 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 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 P25-P50 for phase_3
Started Jun 2009
Typical duration for phase_3
65 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
June 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2010
CompletedResults Posted
Study results publicly available
August 21, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2012
CompletedJanuary 18, 2013
January 1, 2013
1.1 years
May 26, 2009
February 27, 2012
January 14, 2013
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Absolute Mean 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 (5)
Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48
baseline through 48 weeks
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled)
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
Time-to-first Pulmonary Exacerbation 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:
- Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
- Forced expiratory volume in 1 second (FEV1) of 40% to 90% (inclusive) of predicted normal for age, gender, and height at Screening.
- No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
- Willing to use highly effective birth control methods during the study
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
- History of alcohol, medication or illicit drug abuse within one year prior to Day 1
- Abnormal liver function ≥ 3x the upper limit of normal
- Abnormal renal function at Screening
- History of solid organ or hematological transplantation
- Pregnant, planning a pregnancy, breast-feeding, or unwilling to follow contraception requirements
- 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 (65)
University of Alabama
Birmingham, Alabama, 35233-1711, United States
Kaiser Permanente Medical Care Program
Oakland, California, 94611, United States
Cystic Fibrosis Research Office, Stanford University
Palo Alto, California, 94304, United States
Rady Children's Hospital
San Diego, California, 92123-5070, United States
National Jewish Medical and Research Center
Denver, Colorado, 80206, United States
Emory Cystic Fibrosis Center
Atlanta, Georgia, 30322, United States
St. Luke's CF Clinic
Boise, Idaho, 83712, United States
Children's Memorial Hospital
Chicago, Illinois, 60614, United States
Indiana University
Indianapolis, Indiana, 46202, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Johns Hopkins University
Baltimore, Maryland, 21205, 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
Pulmonary, Allergy & Critical Care Medicine, University of Minnesota
Minneapolis, Minnesota, 55455, United States
The Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Washington University
St Louis, Missouri, 63110, United States
Adult Pulmonary/ CF, University of Nebraska Medical Center
Omaha, Nebraska, 68198-5300, United States
Monmouth Medical Center
Long Branch, New Jersey, 07740, United States
Women and Children's Hospital of Buffalo
Buffalo, New York, 14222, United States
Long Island Jewish Medical Center
New Hyde Park, New York, 11042, United States
SUNY Upstate Medical University
Syracuse, New York, 13210, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Pediatric & Pulmonary Division, Rainbow Babies/Case Western
Cleveland, Ohio, 44106, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Toledo Children's Hospital
Toledo, Ohio, 43606, United States
Oregon Health & Sciences University
Portland, Oregon, 97239-3098, United States
Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
East Tennessee Children's Hospital
Knoxville, Tennessee, 37916, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-5735, United States
University of Utah
Salt Lake City, Utah, 84132, United States
University of Virginia
Charlottesville, Virginia, 22908, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Division of Pulmonary and CCM, University of Washington
Seattle, Washington, 98195-6522, United States
West Virginia University
Morgantown, West Virginia, 26506, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
The Children's Hospital Westmead
Westmead, New South Wales, 2145, Australia
The Prince Charles Hospital
Chermside, Queensland, 4032, Australia
Royal Children's Hospital Brisbane
Herston, Queensland, 4026, Australia
Mater Adult Hospital
South Brisbane, Queensland, 4101, Australia
Royal Children's Hospital Melbourne
Parkville, Victoria, 3052, Australia
Lung Institute of Western Australia
Nedlands, Western Australia, 6009, Australia
Princess Margaret Hospital for Children
Subiaco, Western Australia, 6008, Australia
Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, B3H 3A7, Canada
St. Michael's Hospital
Toronto, Ontario, M5B 1W8, Canada
CF Center, Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
Montreal Children's Hospital - MUHC
Montreal, Quebec, H3H 1P3, Canada
FN Motol
Prague, 15006, Czechia
Hopital Cochin
Paris, 75014, France
Hopital Necker
Paris, 75015, France
Centre de Perharidy
Roscoff, 29684, 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
Klinikum der LMU München, Dr. von Haunersches Kinderspital (CHA)
Munich, 80337, Germany
Universitäts-Kinderklinik Würzburg
Würzburg, 97080, Germany
Cork University Hospital
Cork, Ireland
Our Lady's Children's Hospital
Dublin, 12, Ireland
The National Children's Hospital
Dublin, 24, Ireland
St. Vincent's University Hospital
Dublin, 4, Ireland
Beaumont Hospital
Dublin, 9, Ireland
Belfast City Hospital
Belfast, Northern Ireland, BT9 7AB, United Kingdom
Imperial College London
London, SW3 6LR, United Kingdom
Related Publications (4)
Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordonez C, Elborn JS; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185.
PMID: 22047557BACKGROUNDFlume PA, Wainwright CE, Elizabeth Tullis D, Rodriguez S, Niknian M, Higgins M, Davies JC, Wagener JS. Recovery of lung function following a pulmonary exacerbation in patients with cystic fibrosis and the G551D-CFTR mutation treated with ivacaftor. J Cyst Fibros. 2018 Jan;17(1):83-88. doi: 10.1016/j.jcf.2017.06.002. Epub 2017 Jun 24.
PMID: 28651844DERIVEDSolem CT, Vera-Llonch M, Liu S, Botteman M, Castiglione B. Impact of pulmonary exacerbations and lung function on generic health-related quality of life in patients with cystic fibrosis. Health Qual Life Outcomes. 2016 Apr 21;14:63. doi: 10.1186/s12955-016-0465-z.
PMID: 27097977DERIVEDQuittner A, Suthoff E, Rendas-Baum R, Bayliss MS, Sermet-Gaudelus I, Castiglione B, Vera-Llonch M. Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial. Health Qual Life Outcomes. 2015 Jul 2;13:93. doi: 10.1186/s12955-015-0293-6.
PMID: 26135562DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex
Study Officials
- PRINCIPAL INVESTIGATOR
Bonnie W. Ramsey, MD
Children's Hospital and Regional Medical Center, Seattle, Washington, USA
- PRINCIPAL INVESTIGATOR
Stuart Elborn, MD
Respiratory Medicine Group, Queen's University of Belfast, Belfast, Northern Ireland, UK
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
June 1, 2009
Primary Completion
July 1, 2010
Study Completion
November 1, 2012
Last Updated
January 18, 2013
Results First Posted
August 21, 2012
Record last verified: 2013-01