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 trial that will evaluate the clinical benefit of ataluren in adult
and pediatric participants with CF due to a nonsense mutation. The main goals of the study
are to understand whether ataluren can improve pulmonary function and whether the drug can
safely be given for a long period of time. The study will also assess the effects of ataluren
on CF pulmonary exacerbation frequency, cough frequency, health-related quality of life,
antibiotic use for CF-related infections, CF-related disruptions to daily living, body
weight, and CF pathophysiology.
Phase:
Phase 3
Details
Lead Sponsor:
PTC Therapeutics
Collaborators:
Cystic Fibrosis Foundation Cystic Fibrosis Foundation Therapeutics