Efficacy and Safety of Oral PTC124 in Cystic Fibrosis
Research type
Research Study
Full title
A Phase 3 Efficacy and Safety of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
IRAS ID
6979
Contact name
Kevin Southern
Sponsor organisation
PTC Therapeutics
Eudract number
2008-003924-52
Clinicaltrials.gov Identifier
Research summary
Cystic fibrosis (CF) is a disease caused by a mutation (error) in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR), a protein that helps to maintain normal water balance in the mucous of the lungs and other organs. In about 10% of people with CF, a specific mutation type, called a nonsense mutation (premature stop mutation), causes the production of the CFTR protein to stop too early. This makes the protein too short to work properly. This research study will test a new drug called PTC124. Previous studies in adults and children with nonsense mutation caused CF, have shown that PTC124 allows read-through of the premature stop which can result in the production of full-length CFTR protein. This study will test whether PTC124 can: improve lung function, reduce CF symptoms, reduce the number of hospitalisations and use of antibiotics for CF-related lung infections, reduce how often participants cough, improve the participants overall quality of life, and whether the drug is safe for children and adults to take as a long-term treatment.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
09/H1002/59
Date of REC Opinion
26 Aug 2009
REC opinion
Further Information Favourable Opinion