drugset / Trial / NCT00234663
PTC124 for Cystic Fibrosis
Phase 2
Completed
24 enrolled
PTC Therapeutics
Cystic Fibrosis Foundation · collabFDA Office of Orphan Products Development · collab
Non-randomizedSingle-groupOpen-labelTreatment
Summary
In some patients 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. PTC124 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 PTC124 can safely increase functional CFTR protein in the cells of patients with CF due to a nonsense mutation.
Timeline
- Start
- 2005-09
- Primary completion
- 2006-08
- Completion
- 2006-08
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Ataluren | Small molecule | 8 mg/kg | Oral |
| Subject | Ataluren | Small molecule | 20 mg/kg | Oral |