Extension Study of Ataluren (PTC124) in Cystic Fibrosis
Status:
Completed
Trial end date:
2013-12-02
Target enrollment:
Participant gender:
Summary
Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the
cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of
mutation called a nonsense (premature stop codon) mutation is the cause of CF in
approximately 10% of patients with the disease. Ataluren is an orally delivered
investigational drug that has the potential to overcome the effects of the nonsense mutation.
This study is a Phase 3 extension trial that will evaluate the long-term safety of ataluren
in adult and pediatric participants with nonsense mutation CF (nmCF), as determined by
adverse events and laboratory abnormalities. The study will also assess changes in pulmonary
function, CF pulmonary exacerbations, health-related quality of life, antibiotic use for
CF-related infections, CF-related disruptions to daily living, body weight, and CF
pathophysiology. Funding source for this study is the FDA OOPD.
Phase:
Phase 3
Details
Lead Sponsor:
PTC Therapeutics
Collaborators:
Cystic Fibrosis Foundation Cystic Fibrosis Foundation Therapeutics