CPET in CF Patients With One G551D Mutation Taking VX770
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01937325|
Recruitment Status : Unknown
Verified July 2016 by John Wilson, The Alfred.
Recruitment status was: Active, not recruiting
First Posted : September 9, 2013
Last Update Posted : July 25, 2016
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: ivacaftor Drug: placebo||Phase 4|
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 :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Double (Investigator, Outcomes Assessor)|
|Primary Purpose:||Basic Science|
|Official Title:||Airway Infection, Inflammatory Markers and Exercise Capacity in Patients With Cystic Fibrosis and at Least One G551D Mutation Taking VX770 (Ivacaftor)|
|Study Start Date :||February 2014|
|Actual Primary Completion Date :||December 2014|
|Estimated Study Completion Date :||December 2016|
Active Comparator: Ivacaftor
150mg orally twice daily
Other Name: Kalydeco
Placebo Comparator: Placebo
Other Name: ivacaftor matched placebo
- exercise capacity [ Time Frame: one month, 3 months ]Respiratory exercise testing, including spirometry and V02 max.
- 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
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01937325
|Melbourne, Victoria, Australia, 3004|
|Principal Investigator:||John Wilson||Alfred Health and Monash University|