Spots Global Cancer Trial Database for Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

The following info and data is provided "as is" to help patients around the globe.
We do not endorse or review these studies in any way.

Trial Identification

Brief Title: Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

Official Title: 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)

Study ID: NCT02562066

Conditions

Myasthenic Syndromes, Congenital

Interventions

amifampridine phosphate

Placebo

Study Description

Brief 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.