Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12610000491066
Ethics application status
Approved
Date submitted
8/06/2010
Date registered
15/06/2010
Date last updated
24/06/2011
Type of registration
Prospectively registered

Titles & IDs
Public title
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] 251972 0
ClinicalTrials.gov: NCT01117012
Universal Trial Number (UTN)
Trial acronym
PERSIST
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 257535 0
Condition category
Condition code
Human Genetics and Inherited Disorders 257698 257698 0 0
Cystic fibrosis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
VX-770 150mg, oral tablets, twice per day, for the sooner of approximately 96 weeks or until VX 770 is commercially available in each respective country
Intervention code [1] 256630 0
Treatment: Drugs
Comparator / control treatment
Not applicable
Control group
Uncontrolled

Outcomes
Primary outcome [1] 258599 0
To evaluate the safety of long-term VX-770 treatment in subjects with Cystic Fibrosis.as assessed by standard digital electrocardiogram(ECG), blood analysis, coagulation studies, urinalysis, vital signs, physical examination and patient reporting of any adverse event (e.g. gastrointestinal disturbance, nausea, cough, sinus pain, sore throat, etc.)
Timepoint [1] 258599 0
After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.
Secondary outcome [1] 264492 0
Rate of decline in percent predicted Forced Expiratory Volume in 1 second (FEV1), measured by spirometry.
Timepoint [1] 264492 0
After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.
Secondary outcome [2] 264493 0
Absolute change from Day 1 of study VX08-770-105 (Study 105, current study) in FEV1, measured by spirometry.
Timepoint [2] 264493 0
After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.
Secondary outcome [3] 264494 0
Absolute change from Day 1 of previous VX-770 study in FEV1, measured by spirometry.
[Study 102, NCT00909532]
[Study 103, NCT00909727]
Timepoint [3] 264494 0
After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.
Patients must commence this current trial upon completion of the last day on the initial trial.
Secondary outcome [4] 264495 0
Change from Day 1 of Study 105 (current study) in Cystic Fibrosis Questionnaire-Revised (CFQ-R)
Timepoint [4] 264495 0
After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.
Secondary outcome [5] 264496 0
Change from Day 1 of previous VX-770 study in CFQ-R
Timepoint [5] 264496 0
After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.
Secondary outcome [6] 264497 0
Pulmonary exacerbation, defined as new or change in antibiotic therapy for sinopulmonary signs/sypmtoms.
Assessed through sinopulmonary assessment and review of concomitant medication.
Timepoint [6] 264497 0
Throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.
Secondary outcome [7] 264498 0
Rate of change in weight, measured using calibrated scales.
Timepoint [7] 264498 0
After 96 weeks of treatment as assessed throughout the whole duration of the study at Day 15, weeks 12, 24, 36, 48, 60, 72, 84 and 96 of treatment.

Eligibility
Key inclusion criteria
1. Subjects who have completed the assigned study treatment Study 102 or Study 103. 2. Subjects who are female of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770) 3. Subjects 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. Subjects of childbearing potential and who are sexually active must meet the contraception requirements. 5. Subjects 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. Subjects 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. Subjects 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. Subjects who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements.
4. Subjects taking any inhibitors or inducers of CYP3A4, including certain herbal medications (e.g., St John's Wort) and grapefruit/grapefruit juice.

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



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Date of first participant enrolment
Anticipated
1/07/2010
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
200
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment postcode(s) [1] 2981 0
2145
Recruitment postcode(s) [2] 2982 0
3052
Recruitment postcode(s) [3] 2983 0
4101
Recruitment postcode(s) [4] 2984 0
4029
Recruitment postcode(s) [5] 2985 0
4032
Recruitment postcode(s) [6] 2986 0
6009
Recruitment postcode(s) [7] 2987 0
6008
Recruitment outside Australia
Country [1] 2690 0
Canada
State/province [1] 2690 0
Country [2] 2691 0
Czech Republic
State/province [2] 2691 0
Country [3] 2692 0
France
State/province [3] 2692 0
Country [4] 2693 0
Germany
State/province [4] 2693 0
Country [5] 2694 0
Ireland
State/province [5] 2694 0
Country [6] 2695 0
United Kingdom
State/province [6] 2695 0
Country [7] 2696 0
United States of America
State/province [7] 2696 0

Funding & Sponsors
Funding source category [1] 257114 0
Commercial sector/Industry
Name [1] 257114 0
Vertex Pharmaceutical Incorporated
Country [1] 257114 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
Vertex Pharmaceutical Incorporated
Address
130 Waverly Street
Cambridge
MA 02139
Country
United States of America
Secondary sponsor category [1] 256376 0
Other
Name [1] 256376 0
Omnicare Clinical Research Pty Ltd
Address [1] 256376 0
13-15, Lyonpark Road
North Ryde
NSW 2113
Country [1] 256376 0
Australia

Ethics approval
Ethics application status
Approved

Summary
Brief summary
Study 105 (VX08-770-105) is an open-label, VX-770 treatment extension of Study VX08-770-102 (Study 102) and Study VX08-770-103 (Study 103). This rollover study evaluates the long-term safety and efficacy of treatment with the experimental drug, VX-770, for a duration of up to 96 weeks. Male and female subjects 6 years and older with cystic fibrosis who have completed Study 102 or 103 without major protocol violations are eligible to enrol. This project is an international, multi-centre trial, with subjects from 70 sites worldwide including Australia, Europe and North America.
[Study 102: NCT00909532]
[Study 103: NCT00909727]
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 31272 0
Address 31272 0
Country 31272 0
Phone 31272 0
Fax 31272 0
Email 31272 0
Contact person for public queries
Name 14519 0
Medical Monitor
Address 14519 0
Vertex Pharmaceutical Incorporated
130 Waverly Street
Cambridge
MA 02139
Country 14519 0
United States of America
Phone 14519 0
+1 617 444 6777
Fax 14519 0
Email 14519 0
[email protected]
Contact person for scientific queries
Name 5447 0
Medical Monitor
Address 5447 0
Vertex Pharmaceutical Incorporated
130 Waverly Street
Cambridge
MA 02139
Country 5447 0
United States of America
Phone 5447 0
+1 617 444 6777
Fax 5447 0
Email 5447 0
[email protected]

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

Documents added manually
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