Ataluren in Nonsense Mutation Dravet Syndrome or CDKL5 Deficiency

Phase 2, randomised, double-blind trial of ataluren in Dravet syndrome and CDKL5 deficiency disorder (CDD) (2 arms), sponsored by NYU Langone Health. Primary endpoints: Percent Change From Baseline in 28-Day Convulsive Seizure Frequency During Ataluren Treatment Period at 12 weeks and Percent Change From Baseline in 28-Day Convulsive Seizure Frequency During Placebo Treatment Period at 12 weeks. Started Nov 2016; status: completed.