Trajectory
Research type
Research Study
Full title
A Longitudinal Study on the Impact of ELX/TEZ/IVA Treatment in Patients With Cystic Fibrosis in the Real World.
IRAS ID
306934
Contact name
Jamie Duckers
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Clinicaltrials.gov Identifier
253669, Parexel Project Number
Duration of Study in the UK
5 years, 9 months, days
Research summary
Cystic fibrosis (CF) is an autosomal recessive, life shortening, and multisystemic disease. Patients with CF have significant disease burden, including chronic lung infections, gastrointestinal problems, CF-related diabetes, and other complex comorbidities. There is currently no cure for CF, and the impact and time commitment of the disease and its management remain substantial. Daily activities require an immense amount of effort while maintaining compliance with treatment, which is an average of 2 hours per day. CFTR modulators are systemic therapies that improve pulmonary and non-pulmonary outcomes and, with extended use, have been shown to modify the course of CF disease by improving survival and reducing the rate of decline in lung function.
A triple combination regimen of CFTR modulators including elexacaftor, tezacaftor, and ivacaftor (ELX/TEZ/IVA) is being developed for patients with CF, including pediatric patients. Currently, ELX/TEZ/IVA is approved in the US and Europe for the treatment of patients with CF who are 12 years of age and older (see product label). However, the impact of ELX/TEZ/IVA on the clinical characteristics and treatment burden on patients after initiating treatment with ELX/TEZ/IVA in a real-world setting has not been fully assessed.
This observational study will describe the changes in clinical characteristics, treatment burden, and quality of life of patients with CF after initiation of ELX/TEZ/IVA in a real world setting using EMRs.REC name
South Central - Hampshire B Research Ethics Committee
REC reference
22/SC/0019
Date of REC Opinion
16 Mar 2022
REC opinion
Further Information Favourable Opinion