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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
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
Secondary ID [1] 0 0
VX08-770-102
Universal Trial Number (UTN)
Trial acronym
STRIVE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24
Timepoint [1] 0 0
baseline through 24 weeks
Secondary outcome [1] 0 0
Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48
Timepoint [1] 0 0
baseline through 48 weeks
Secondary outcome [2] 0 0
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled)
Timepoint [2] 0 0
baseline through 24 weeks and 48 weeks
Secondary outcome [3] 0 0
Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48
Timepoint [3] 0 0
baseline through 24 weeks and 48 weeks
Secondary outcome [4] 0 0
Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48
Timepoint [4] 0 0
baseline through 24 weeks and 48 weeks
Secondary outcome [5] 0 0
Absolute Change From Baseline in Weight at Week 24 and Week 48
Timepoint [5] 0 0
baseline to 24 weeks and 48 weeks

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
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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)

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2009
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/11/2012
Sample size
Target
Accrual to date
Final
167
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
The Children's Hospital Westmead - Westmead
Recruitment hospital [2] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [3] 0 0
Royal Children's Hospital Brisbane - Herston
Recruitment hospital [4] 0 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [5] 0 0
Royal Children's Hospital Melbourne - Parkville
Recruitment hospital [6] 0 0
Lung Institute of Western Australia - Nedlands
Recruitment hospital [7] 0 0
Princess Margaret Hospital for Children - Subiaco
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4032 - Chermside
Recruitment postcode(s) [3] 0 0
4026 - Herston
Recruitment postcode(s) [4] 0 0
4101 - South Brisbane
Recruitment postcode(s) [5] 0 0
3052 - Parkville
Recruitment postcode(s) [6] 0 0
6009 - Nedlands
Recruitment postcode(s) [7] 0 0
6008 - Subiaco
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
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California
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Colorado
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Georgia
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Idaho
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Illinois
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Indiana
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Iowa
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Maryland
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Massachusetts
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Michigan
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Minnesota
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Missouri
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Nebraska
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New Jersey
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New York
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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Tennessee
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Utah
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Virginia
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Washington
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West Virginia
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Wisconsin
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Canada
State/province [27] 0 0
Nova Scotia
Country [28] 0 0
Canada
State/province [28] 0 0
Ontario
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Canada
State/province [29] 0 0
Quebec
Country [30] 0 0
Czech Republic
State/province [30] 0 0
Prague
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France
State/province [31] 0 0
Paris
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France
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Roscoff
Country [33] 0 0
Germany
State/province [33] 0 0
Erlangen
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Germany
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Jena
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Germany
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Munich
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Germany
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Wurzburg
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Ireland
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Cork
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Ireland
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Dublin
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United Kingdom
State/province [39] 0 0
Northern Ireland
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United Kingdom
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London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Vertex Pharmaceuticals Incorporated
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Cystic Fibrosis Foundation
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Bonnie W. Ramsey, MD
Address 0 0
Children's Hospital and Regional Medical Center, Seattle, Washington, USA
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
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