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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT01117012
Registration number
NCT01117012
Ethics application status
Date submitted
3/05/2010
Date registered
5/05/2010
Date last updated
7/07/2015
Titles & IDs
Public title
Rollover Study of VX-770 in Cystic Fibrosis Subjects
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Scientific title
An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX-770 in Subjects With Cystic Fibrosis
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Secondary ID [1]
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PERSIST
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Secondary ID [2]
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VX08-770-105
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Universal Trial Number (UTN)
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Trial acronym
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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
Experimental: VX-770 - VX-770 (ivacaftor) 150 milligram (mg) tablet orally twice daily (q12h).
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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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Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Assessment method [1]
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Adverse Event: any untoward medical occurrence in a participant during the study, including any unfavorable and unintended sign, symptom, or disease whether or not it was considered to be study drug related. This included any newly occurring event or previous condition that increased in severity or frequency after obtaining informed consent and assent (where applicable). SAE: medical event or condition, which resulted in any of following, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event. Non-Serious AEs included all AEs except SAEs.
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Timepoint [1]
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Study 105: Day 1 up to Week 168
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Secondary outcome [1]
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Annualized Rate of Decline From Study 105 Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 96
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Assessment method [1]
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as Study 105 Day 15.
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Timepoint [1]
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Study 105: Baseline through Week 96
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Secondary outcome [2]
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Absolute Change From Study 105 Baseline in Percent Predicted FEV1 Through Week 96
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Assessment method [2]
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in Study 105. Absolute Change at Week 48 and Week 96 are reported.
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Timepoint [2]
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Study 105: Baseline through Week 96
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Secondary outcome [3]
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Absolute Change From Study 105 Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 96
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Assessment method [3]
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The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for subjects with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Absolute Change at Week 48 and Week 96 are reported.
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Timepoint [3]
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Study 105: Baseline through Week 96
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Secondary outcome [4]
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Annualized Pulmonary Exacerbation Event Rate
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Assessment method [4]
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Annualized event rate was calculated by regression with negative binomial distribution.
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Timepoint [4]
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Study 105: Day 1 through Week 96
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Secondary outcome [5]
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Annualized Duration of Pulmonary Exacerbation Events
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Assessment method [5]
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Timepoint [5]
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Study 105: Day 1 through Week 96
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Secondary outcome [6]
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Absolute Change From Study 105 Baseline in Weight Through Week 96
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Assessment method [6]
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Weight is a measurement of nutritional status. Absolute change in weight, measured in kilograms (kg), at Week 48 and Week 96 are reported.
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Timepoint [6]
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Study 105: Baseline through Week 96
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Eligibility
Key inclusion criteria
1. Participants who have completed the assigned study treatment in Study 102 or Study 103
2. Participants who are females of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770)
3. Participants who are able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, as judged by the investigator
4. Participants of child bearing potential and who are sexually active must meet the contraception requirements
5. Participants must sign the informed consent form (ICF), and where appropriate, assent must be obtained
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Minimum age
6
Years
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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
1. Participants with a history of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject
2. Participants with a history of study treatment intolerance as observed in their previous VX-770 study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject
3. Participants who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements
4. Participants taking any inhibitors or inducers of Cytochrome P450 3A4 (CYP3A4), including certain herbal medications (for example, St. John's Wort) and grapefruit/grapefruit juice
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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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
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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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
1/07/2010
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/05/2014
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Sample size
Target
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Accrual to date
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Final
192
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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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- Westmead
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Recruitment hospital [2]
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- Chermside
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- Herston
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- South Brisbane
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- Parkville
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- Nedlands
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- Subiaco
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- Westmead
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- Chermside
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- Herston
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- South Brisbane
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- Parkville
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- Nedlands
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Recruitment postcode(s) [7]
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- 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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Colorado
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Illinois
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West Virginia
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France
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Paris
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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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Germany
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Munich
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Wurzburg
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Cork
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Dublin
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Northern Ireland
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United Kingdom
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London
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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 primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF). The secondary objective of the study was to evaluate the efficacy of long-term VX-770 treatment in subjects with CF.
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Trial website
https://clinicaltrials.gov/study/NCT01117012
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Trial related presentations / publications
McKone EF, Borowitz D, Drevinek P, Griese M, Konstan MW, Wainwright C, Ratjen F, Sermet-Gaudelus I, Plant B, Munck A, Jiang Y, Gilmartin G, Davies JC; VX08-770-105 (PERSIST) Study Group. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST). Lancet Respir Med. 2014 Nov;2(11):902-910. doi: 10.1016/S2213-2600(14)70218-8. Epub 2014 Oct 9.
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Public notes
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Contacts
Principal investigator
Name
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Edward McKone, MD
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Address
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St. Vincent's University Hospital
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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/NCT01117012
Download to PDF