Expanded Access Study Amifampridine Phosphate in Congenital Myasthenic Syndrome (CMS)
No longer available
Catalyst Pharmaceuticals, Inc.
1969-12-31
The primary objective of the study is:
• To provide patients with CMSaccess to amifampridine phosphate therapy until the product
becomes commercially available or development is discontinued.
The secondary objective of the study is:
• To assess the long-term safety of amifampridine phosphate in patients with CMS
Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes
Completed
Catalyst Pharmaceuticals, Inc.
Phase 3
2016-01-01
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.
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