A Phase 3, Double-blind, Outpatient Crossover Study to Evaluate the Efficacy and Safety of Amifampridine Phosphate (3,4 Diaminopyridine Phosphate) in Patients With Congenital Myasthenic Syndromes (CMS)

Trial Profile

A Phase 3, Double-blind, Outpatient Crossover Study to Evaluate the Efficacy and Safety of Amifampridine Phosphate (3,4 Diaminopyridine Phosphate) in Patients With Congenital Myasthenic Syndromes (CMS)

Recruiting
Phase of Trial: Phase III

Latest Information Update: 09 Aug 2017

At a glance

  • Drugs Amifampridine (Primary)
  • Indications Congenital myasthenic syndromes
  • Focus Therapeutic Use
  • Sponsors Catalyst Pharmaceutical Partners
  • Most Recent Events

    • 09 Aug 2017 According to Catalyst Pharmaceuticals media release, top-line results from CMS trial are expected in the first half of 2018.
    • 09 Aug 2016 According to Catalyst Pharmaceuticals media release, FDA is currently reviewing amended protocol and statistical analysis plans. Top-line data from this trial expected in the second half of 2017. Based on the favorable data from this trial company plans to include Congenital Myasthenic Syndromes in NDA resubmission or as a supplement to the NDA. Patients from this trial will be eligible to enroll in an Expanded Access Program at the completion of this study.
    • 26 Jul 2016 Age criteria has been changed from 2-17 years to 2-70 years.
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