Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
Status:
Completed
Trial end date:
2021-02-27
Target enrollment:
Participant gender:
Summary
This is a phase 2, crossover study of Ataluren for the treatment of nonsense mutation Dravet
syndrome or cyclin-dependent kinase-like 5 (CDKL5) deficiency, resulting in drug-resistant
epilepsy. Patients will receive 12 weeks of ataluren or placebo during each treatment period.
Treatment Period 1 will be followed by a 4-week Washout Period. Based on ataluren PK and
pharmacodynamic data, the 4-week washout period is deemed an appropriate length of time to
eliminate any ataluren drug effects. Following the Washout Period, patients will crossover to
receive the opposite treatment during Treatment Period 2 as follows: Patients receiving
ataluren during Treatment Period 1 will receive placebo during Treatment Period 2. Patients
receiving placebo during Treatment Period 1 will receive ataluren during Treatment Period 2.
Phase:
Phase 2
Details
Lead Sponsor:
New York University School of Medicine NYU Langone Health