A Ph3 Extension Study of Ataluren (PTC124) in Patients with nm-CF

• Research type

  Research Study

• Full title

  A Phase 3 Extension Study of Ataluren (PTC124®) in Patients With Nonsense Mutation Cystic Fibrosis

• IRAS ID

  185377

• Contact name

  Gordon MacGregor

• Contact email

  gordon.macgregor@ggc.scot.nhs.uk

• Sponsor organisation

  PTC Therapeutics, Inc.

• Eudract number

  2014-005355-83

• Clinicaltrials.gov Identifier

  NCT02456103

• Duration of Study in the UK

  2 years, 1 months, 1 days

• 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 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 the results from clinical trials in ataluren have shown that the drug appears to be safe and well tolerated.

  There is currently an ongoing clinical trial (PTC124-GD-021-CF) which assesses the safety and effectiveness of ataluren in 208 patients worldwide with nmCF who are 6 years or older. After 48 weeks of treatment with ataluren or placebo, patients who successfully complete this study will have the chance to go onto this extension study (PTC124-GD-021e-CF).
  There will be 9 study visits. Study procedures will include spirometry, blood tests, physical examination, vital signs, sputum, 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

  16/WS/0041

• Date of REC Opinion

  22 Mar 2016

• REC opinion

  Further Information Favourable Opinion