A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
Status:
Completed
Trial end date:
2008-02-29
Target enrollment:
Participant gender:
Summary
In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation
(premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator
(CFTR) protein. Ataluren has been shown to partially restore CFTR production in animals with
CF due to a nonsense mutation. The main purpose of this study is to understand whether
ataluren can safely increase functional CFTR protein in the cells of participants with CF due
to a nonsense mutation.