None listed
Conditions
Brief summary
Our aim was to determine a sensitive clinical measure of change, in patients with CF and severe airways disease as measured by an FEV1<40% when stable, following treatment with Lumacaftor/Ivacaftor. We assessed response with spirometry and compared this to changes seen in exercise capacity as measured by the six minute walk test (6MWT), the nitrogen multi-breath washout test, the carbon monoxide gas transfer factor and lung volumes measured by plethysmography.
Interventions
Participants received Lumacaftor 400mg and Ivacaftor 250mg twice daily for 12 months. They received these as part of treatment, not for the study.Participation did not require any additional investigations or review. The data from the medical record was reviewed. All investigations were done as part of usual care.
Sponsors
John Hunter Hospital
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
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.
Exclusion criteria
Unable to participate in the measures required.
Outcome results
None listed