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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. 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
Information provided by (Responsible Party):
John Wilson, The Alfred

Brief Summary:
Ivacaftor will restore CFTR function in treated CF patients with the G551D mutation. Improvement in ventilation, salt balance and well-being will contribute to better exercise capacity at all levels of lung function. While potential improvements may be variable across the spectrum of lung function, even small gains at low levels of FEV1 may have significant benefit for some subjects.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: ivacaftor Drug: placebo Phase 4

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: Randomized
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

Arm Intervention/treatment
Active Comparator: Ivacaftor
150mg orally twice daily
Drug: ivacaftor
active arm
Other Name: Kalydeco

Placebo Comparator: Placebo
Matching placebo
Drug: placebo
active arm
Other Name: ivacaftor matched placebo

Primary Outcome Measures :
  1. exercise capacity [ Time Frame: one month, 3 months ]
    Respiratory exercise testing, including spirometry and V02 max.

Secondary Outcome Measures :
  1. 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

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01937325

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Australia, Victoria
The Alfred
Melbourne, Victoria, Australia, 3004
Sponsors and Collaborators
The Alfred
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Principal Investigator: John Wilson Alfred Health and Monash University

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: John Wilson, Head CF Service, The Alfred Identifier: NCT01937325    
Other Study ID Numbers: CPET in CF
First Posted: September 9, 2013    Key Record Dates
Last Update Posted: July 25, 2016
Last Verified: July 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by John Wilson, The Alfred:
Eligible CF patients with G551D gene mutation
Additional relevant MeSH terms:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action