Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes
Status:
Completed
Trial end date:
2019-10-01
Target enrollment:
Participant gender:
Summary
This randomized, double-blind, controlled, outpatient two-period, two-treatment crossover
study is designed to evaluate the efficacy and safety of amifampridine phosphate in patients
(ages 2 and above) diagnosed with certain genetic subtypes of CMS and demonstrated open label
(amifampridine phosphate) or history of sustained amifampridine benefit from treatment.