CPET in CF Patients With One G551D Mutation Taking VX770

• Status: Unknown status
• Trial end date: 2016-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: The Alfred
• Treatments: Ivacaftor

Criteria

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).