Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

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.

Timeline

Start

2016-01

Primary completion

2019-08

Completion

2019-10

Drugs

Indications