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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
Scientific title
An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX-770 in Subjects With Cystic Fibrosis
Secondary ID [1] 0 0
PERSIST
Secondary ID [2] 0 0
VX08-770-105
Universal Trial Number (UTN)
Trial acronym
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
Experimental: VX-770 - VX-770 (ivacaftor) 150 milligram (mg) tablet orally twice daily (q12h).
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
Study 105: Day 1 up to Week 168
Secondary outcome [1] 0 0
Annualized Rate of Decline From Study 105 Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 96
Timepoint [1] 0 0
Study 105: Baseline through Week 96
Secondary outcome [2] 0 0
Absolute Change From Study 105 Baseline in Percent Predicted FEV1 Through Week 96
Timepoint [2] 0 0
Study 105: Baseline through Week 96
Secondary outcome [3] 0 0
Absolute Change From Study 105 Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 96
Timepoint [3] 0 0
Study 105: Baseline through Week 96
Secondary outcome [4] 0 0
Annualized Pulmonary Exacerbation Event Rate
Timepoint [4] 0 0
Study 105: Day 1 through Week 96
Secondary outcome [5] 0 0
Annualized Duration of Pulmonary Exacerbation Events
Timepoint [5] 0 0
Study 105: Day 1 through Week 96
Secondary outcome [6] 0 0
Absolute Change From Study 105 Baseline in Weight Through Week 96
Timepoint [6] 0 0
Study 105: Baseline through Week 96

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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
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
1/07/2010
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/05/2014
Sample size
Target
Accrual to date
Final
192
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
- Westmead
Recruitment hospital [2] 0 0
- Chermside
Recruitment hospital [3] 0 0
- Herston
Recruitment hospital [4] 0 0
- South Brisbane
Recruitment hospital [5] 0 0
- Parkville
Recruitment hospital [6] 0 0
- Nedlands
Recruitment hospital [7] 0 0
- Subiaco
Recruitment postcode(s) [1] 0 0
- Westmead
Recruitment postcode(s) [2] 0 0
- Chermside
Recruitment postcode(s) [3] 0 0
- Herston
Recruitment postcode(s) [4] 0 0
- South Brisbane
Recruitment postcode(s) [5] 0 0
- Parkville
Recruitment postcode(s) [6] 0 0
- Nedlands
Recruitment postcode(s) [7] 0 0
- Subiaco
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Idaho
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Indiana
Country [8] 0 0
United States of America
State/province [8] 0 0
Iowa
Country [9] 0 0
United States of America
State/province [9] 0 0
Maryland
Country [10] 0 0
United States of America
State/province [10] 0 0
Massachusetts
Country [11] 0 0
United States of America
State/province [11] 0 0
Michigan
Country [12] 0 0
United States of America
State/province [12] 0 0
Minnesota
Country [13] 0 0
United States of America
State/province [13] 0 0
Missouri
Country [14] 0 0
United States of America
State/province [14] 0 0
Nebraska
Country [15] 0 0
United States of America
State/province [15] 0 0
New Jersey
Country [16] 0 0
United States of America
State/province [16] 0 0
New York
Country [17] 0 0
United States of America
State/province [17] 0 0
Ohio
Country [18] 0 0
United States of America
State/province [18] 0 0
Oregon
Country [19] 0 0
United States of America
State/province [19] 0 0
Pennsylvania
Country [20] 0 0
United States of America
State/province [20] 0 0
Tennessee
Country [21] 0 0
United States of America
State/province [21] 0 0
Utah
Country [22] 0 0
United States of America
State/province [22] 0 0
Virginia
Country [23] 0 0
United States of America
State/province [23] 0 0
Washington
Country [24] 0 0
United States of America
State/province [24] 0 0
West Virginia
Country [25] 0 0
Canada
State/province [25] 0 0
British Columbia
Country [26] 0 0
Canada
State/province [26] 0 0
Nova Scotia
Country [27] 0 0
Canada
State/province [27] 0 0
Ontario
Country [28] 0 0
Canada
State/province [28] 0 0
Quebec
Country [29] 0 0
Czech Republic
State/province [29] 0 0
Prague
Country [30] 0 0
France
State/province [30] 0 0
Paris
Country [31] 0 0
France
State/province [31] 0 0
Roscoff
Country [32] 0 0
Germany
State/province [32] 0 0
Erlangen
Country [33] 0 0
Germany
State/province [33] 0 0
Jena
Country [34] 0 0
Germany
State/province [34] 0 0
Munich
Country [35] 0 0
Germany
State/province [35] 0 0
Wurzburg
Country [36] 0 0
Ireland
State/province [36] 0 0
Cork
Country [37] 0 0
Ireland
State/province [37] 0 0
Dublin
Country [38] 0 0
United Kingdom
State/province [38] 0 0
Northern Ireland
Country [39] 0 0
United Kingdom
State/province [39] 0 0
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
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.
Trial website
https://clinicaltrials.gov/study/NCT01117012
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.
Public notes

Contacts
Principal investigator
Name 0 0
Edward McKone, MD
Address 0 0
St. Vincent's University Hospital
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01117012