NCT02562066

Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

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)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Catalyst Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 2 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Each patient will participate in an open-label unblinded drug escalation/treatment run-in phase for up to 4 weeks until stable dose and frequency of amifampridine phosphate is achieved for 7 days. After this phase, blinded treatment effect will be assessed in a randomized fashion of continuation or cessation of drug (Placebo) starting with Period I (duration 7 days). Following experimental Period 1, patients will be returned to the stable dose administered at the end of the open-label run-in period for approximately 2 weeks, followed by cross over treatment in Period 2 dosing for 7 days. After completion of Period 2, patients will be eligible for expanded access with restoration of open-label amifampridine phosphate at the same dose and frequency as established in the run in phase of the study.

Conditions

• Myasthenic Syndromes, Congenital

Interventions

Type	Name	Description
DRUG	amifampridine phosphate	Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.
DRUG	Placebo	A placebo equivalent will be provided as tablets indistinguishable from the amifampridine phosphate tablets. The placebo will be administered consistent with the dose regimen of amifampridine phosphate.

Timeline

• Start date: 2016-01-01
• Primary completion: 2019-08-01
• Completion: 2019-10-01
• First posted: 2015-09-29
• Last updated: 2021-04-02
• Results posted: 2021-04-02

Locations

4 sites across 1 country: United States