CPET in CF Patients With One G551D Mutation Taking VX770

NCT01937325 | Unknown Phase 4

• 1 other identifier: CPET in CF
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis

Keywords

Eligible CF patients with G551D gene mutation

Outcome Measures

Primary Outcomes (1)

• exercise capacity

  Respiratory exercise testing, including spirometry and V02 max.

  one month, 3 months

Secondary Outcomes (1)

• Inflammatory profile

  One month, 3 months

Study Arms (2)

Ivacaftor

ACTIVE COMPARATOR

150mg orally twice daily

Drug: ivacaftor

Placebo

PLACEBO COMPARATOR

Matching placebo

Drug: placebo

Interventions

ivacaftor DRUG

active arm

Also known as: Kalydeco

Ivacaftor

placebo DRUG

active arm

Also known as: ivacaftor matched placebo

Placebo

Eligibility Criteria

• Age: 16 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• The Alfred: lead

Study Sites (1)

The Alfred

Melbourne, Victoria, 3004, Australia

Location

Related Publications (1)

• Edgeworth D, Keating D, Ellis M, Button B, Williams E, Clark D, Tierney A, Heritier S, Kotsimbos T, Wilson J. Improvement in exercise duration, lung function and well-being in G551D-cystic fibrosis patients: a double-blind, placebo-controlled, randomized, cross-over study with ivacaftor treatment. Clin Sci (Lond). 2017 Jul 16;131(15):2037-2045. doi: 10.1042/CS20170995. Print 2017 Aug 1.

  PMID: 28611235 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• John Wilson

  Alfred Health and Monash University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: BASIC SCIENCE
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head CF Service

Study Record Dates

• First Submitted: September 4, 2013
• First Posted: September 9, 2013
• Study Start: February 1, 2014
• Primary Completion: December 1, 2014
• Study Completion: December 1, 2016
• Last Updated: July 25, 2016

Record last verified: 2016-07

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)