Phase III extension of Efficacy & Safety of PTC124 in Cystic Fibrosis

• Research type

  Research Study

• Full title

  A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis

• IRAS ID

  56514

• Sponsor organisation

  PTC Therapeutics, Inc

• Eudract number

  2010-019692-30

• Clinicaltrials.gov Identifier

  NCT01140451

• 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 ataluren (PTC124). Previous studies in adults and children with nonsense mutation caused CF, have shown that ataluren allows read-through of the premature stop which can result in the production of full-length CFTR protein.This study will evaluate the long term safety and efficacy of ataluren in patients with nonsense mutation mediated CF (nmCF).

• REC name

  North West - Liverpool Central Research Ethics Committee

• REC reference

  11/NW/0323

• Date of REC Opinion

  21 Jul 2011

• REC opinion

  Further Information Favourable Opinion