Phase 3 trial of ataluren in patients with nonsense mutation CF
Research type
Research Study
Full title
A PHASE 3 EFFICACY AND SAFETY STUDY OF ATALUREN (PTC124®) IN PATIENTS WITH NONSENSE MUTATION CYSTIC FIBROSIS
IRAS ID
155214
Contact name
Gordon MacGregor
Contact email
Sponsor organisation
PTC Therapeutics, Inc.
Eudract number
2013-004581-34
Research summary
Cystic fibrosis (CF) is an orphan disease which is estimated to affect approximately 10,000 people in the UK. CF is a disabling and life-threatening genetic disorder resulting from mutations in the gene responsible for the cystic fibrosis transmembrane conductance regulator (CFTR). The CFTR is a protein which controls chloride transport within the body. The disruption of chloride transport and associated water transport abnormalities results in a build-up of thick mucus in the respiratory tract, pancreas, gastrointestinal tract, sweat glands and other tissues. CF patients suffer chronic respiratory disease, pancreatic and liver abnormalities, intestinal obstruction and reduced fertility. The median life expectancy is approximately 35 years.
Approximately 10% of CF patients have a form of CF due to a nonsense mutation CF (nmCF). Patients with nmCF cannot make a complete CFTR protein. Current available treatments for nmCF are exclusively supportive. Ataluren is a novel investigational drug which works by helping the body to ignore the nonsense mutation and make a complete and functional CFTR protein. So far ataluren has only been tested in clinical trials, but the results have been promising and the drug appears to be safe and well tolerated.
This trial will assess the safety and effectiveness of ataluren in 208 patients worldwide with nmCF who are 6 years or older. It is a double blind placebo controlled trial. Study drug will be taken orally 3 times daily. After 48 weeks of treatment with ataluren or placebo, participants will have the option to receive ataluren in an open label extension study.
There will be 9 study visits plus long-term follow up. Study procedures will include spirometry, blood tests, physical examination, vital signs, sputum, stool and urine sample collection, ECG, questionnaires and renal ultrasound.
The study is sponsored by PTC Therapeutics Inc.
REC name
West of Scotland REC 1
REC reference
14/WS/1021
Date of REC Opinion
22 Jul 2014
REC opinion
Further Information Favourable Opinion