- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01937325
CPET in CF Patients With One G551D Mutation Taking VX770
Airway Infection, Inflammatory Markers and Exercise Capacity in Patients With Cystic Fibrosis and at Least One G551D Mutation Taking VX770 (Ivacaftor)
Study Overview
Detailed Description
The Alfred CF Service is ready to initiate therapy with VX-770 in it's cohort. Investigators therefore have an opportunity to examine correlates between improvements in lung function, exercise capacity, inflammatory markers and indices of wellbeing in CF that may not be repeatable in a single cohort.
Investigators propose a double-blind, placebo-controlled cross-over study of 20 patients currently awaiting initiation of ivacaftor therapy. Patients enrolled in the study will be asked to undergo screening (day -28), and baseline assessments (day 0) and re-assessment studies (day 28) after treatment period 1, followed by baseline assessment at the beginning of treatment period 2 (day 56) and at the conclusion of treatment period 2 (day 84). A further assessment (day 224) will be performed 140 (+/-7) days following commencement on open label Ivacaftor.
After satisfying eligibility criteria, subjects will be randomly assigned to initial active treatment or placebo following a 4 week run-in period. After completion of period 1 and a 4 week washout period, subjects will cross-over to the alternative treatment. After 4 weeks of period 2, subjects will undergo final assessment as shown in the diagram below. However participants choosing not to continue taking ivacaftor in a Named Patient Program or another similar program run by Vertex Pharmaceuticals, Inc., ('Vertex'), at the end of the study period will be required to undergo a Safety Follow-Up visit 28 days after the final dose of study drug. Safety Follow-Up assessments will not include the CPET.
Study Type
Enrollment (Actual)
Phase
- Phase 4
Contacts and Locations
Study Locations
-
-
Victoria
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Melbourne, Victoria, Australia, 3004
- The Alfred
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- All participants will have CF proven based on established criteria (sweat test, genotype and phenotype).
- All participants will have at least one copy of the G551D mutation.
- All will be able to perform an exercise study and complete study questionnaires and assessments.
- Age range will be between 16 and 75 years of age.
- Lung function inclusion will be above 25% predicted FEV1.
Exclusion Criteria:
- Participants will not be included if they are unable to complete study assessments or have had a known adverse reaction to Ivacaftor.
- Female participants will be excluded if found to return a positive pregnancy test at screening.
- Participants will be excluded if using St. John's Wort or rifampicin (strong CYP3A inducers).
- Participants with significant liver dysfunction will be excluded (ALT or ALT above 5 times upper limit of normal).
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Basic Science
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Double
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: Placebo
Matching placebo
|
active arm
Other Names:
|
Active Comparator: Ivacaftor
150mg orally twice daily
|
active arm
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
exercise capacity
Time Frame: one month, 3 months
|
Respiratory exercise testing, including spirometry and V02 max.
|
one month, 3 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Inflammatory profile
Time Frame: One month, 3 months
|
Cytokine levels (IL-1β, IL-6, TNFα, IL-8, VEGF & Activin A) determined using cytometric bead analysis and / or ELISA
|
One month, 3 months
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: John Wilson, Alfred Health and Monash University
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- CPET in CF
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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