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.
Phase 3 Study to Evaluate Efficacy of Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS)
Completed
Catalyst Pharmaceuticals, Inc.
Phase 3
2016-11-01
This study evaluates the effect of withdrawing amifampridine phosphate treatment from
patients with LEMS. One half of the patients will continue to receive amifampridine phosphate
and the other half will receive placebo, during this double-blind study.
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