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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT00909532
Registration number
NCT00909532
Ethics application status
Date submitted
26/05/2009
Date registered
28/05/2009
Date last updated
18/01/2013
Titles & IDs
Public title
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation
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Scientific title
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
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Secondary ID [1]
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VX08-770-102
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Universal Trial Number (UTN)
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Trial acronym
STRIVE
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Cystic fibrosis
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Respiratory
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Other respiratory disorders / diseases
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Oral and Gastrointestinal
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Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
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Inflammatory and Immune System
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Connective tissue diseases
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Inflammatory and Immune System
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Other inflammatory or immune system disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Ivacaftor
Treatment: Drugs - Placebo
Placebo comparator: Placebo - Subjects who received placebo every 12 hours (q12h) for up to 48 weeks.
Experimental: 150 mg Ivacaftor q12h - Subjects who received 150 mg of ivacaftor q12h for up to 48 weeks.
Treatment: Drugs: Ivacaftor
150-mg tablets given orally q12h for up to 48 weeks
Treatment: Drugs: Placebo
Tablet given orally q12h for up to 48 weeks
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24
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Assessment method [1]
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Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
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Timepoint [1]
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baseline through 24 weeks
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Secondary outcome [1]
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Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48
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Assessment method [1]
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Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
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Timepoint [1]
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baseline through 48 weeks
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Secondary outcome [2]
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Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled)
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Assessment method [2]
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The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID).
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Timepoint [2]
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baseline through 24 weeks and 48 weeks
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Secondary outcome [3]
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Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48
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Assessment method [3]
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The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity.
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Timepoint [3]
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baseline through 24 weeks and 48 weeks
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Secondary outcome [4]
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Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48
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Assessment method [4]
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Pulmonary exacerbation was defined as a change in antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of signs/symptoms such as change in sputum; new or increased hemoptysis; increased cough or dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees C; anorexia or weight loss; sinus pain/tenderness and discharge; change in physical examination of the chest; decreased pulmonary function by 10%; and radiographic changes indicative of pulmonary infection.
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Timepoint [4]
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baseline through 24 weeks and 48 weeks
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Secondary outcome [5]
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Absolute Change From Baseline in Weight at Week 24 and Week 48
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Assessment method [5]
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As malnutrition is common in patients with cystic fibrosis (CF) because of increased energy expenditures due to lung disease and fat malabsorption, body weight is an important clinical measure of nutritional status.
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Timepoint [5]
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baseline to 24 weeks and 48 weeks
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Eligibility
Key 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 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
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Minimum age
12
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* 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)
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/06/2009
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Date of last participant enrolment
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Actual
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Date of last data collection
Anticipated
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Actual
1/11/2012
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Sample size
Target
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Accrual to date
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Final
167
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
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Recruitment hospital [1]
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The Children's Hospital Westmead - Westmead
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The Prince Charles Hospital - Chermside
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Royal Children's Hospital Brisbane - Herston
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Mater Adult Hospital - South Brisbane
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Royal Children's Hospital Melbourne - Parkville
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Lung Institute of Western Australia - Nedlands
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Princess Margaret Hospital for Children - Subiaco
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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4032 - Chermside
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Recruitment postcode(s) [3]
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4026 - Herston
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Recruitment postcode(s) [4]
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4101 - South Brisbane
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Recruitment postcode(s) [5]
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3052 - Parkville
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Recruitment postcode(s) [6]
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6009 - Nedlands
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Recruitment postcode(s) [7]
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6008 - Subiaco
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Recruitment outside Australia
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United States of America
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Alabama
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California
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Prague
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France
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Roscoff
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Germany
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Erlangen
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Germany
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Jena
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Vertex Pharmaceuticals Incorporated
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Address
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Other collaborator category [1]
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Other
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Name [1]
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Cystic Fibrosis Foundation
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Address [1]
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT00909532
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Trial related presentations / publications
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. Flume 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. Solem 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. Quittner 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.
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Public notes
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Contacts
Principal investigator
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Bonnie W. Ramsey, MD
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Address
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Children's Hospital and Regional Medical Center, Seattle, Washington, USA
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT00909532
Download to PDF