Observation effect of Ivacaftor/lumacaftor on lung function and exercise tolerance in subjects with Cystic Fibrosis and severe lung disease
- Conditions
- Cystic FibrosisHuman Genetics and Inherited Disorders - Cystic fibrosis
- Registration Number
- ACTRN12619000708156
- Lead Sponsor
- John Hunter Hospital
- Brief Summary
Treatment of people living with CF and 2 copies of the delta F 508 gene with Orkambi (lumacaftor/Ivacaftor) has been shown to improve outcomes in those with milder forms of lung disease. In those excluded from the trials with severe disease, this is not known. We carefully assessed the effect of treatment on 10 individuals with severe lung disease and CF. WE showed improvments in exercise tolerance (6 minute walk test) and some effects on lung function, though this took up to 24 weeks to be demonstrated.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 20
Diagnosis of Cystic fibrosis, aged 18years, and homozygous for Phe508del mutation, eligible for access to LUM/IVA through a compassionate access programme provided by Vertex. To be eligible they had to have an FEV1? 40% when clinically stable, or experienced a 20% or more fall in FEV1, or had been referred for lung transplantation assessment.
Unable to participate in the measures required.
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method