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Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

NCT02562066 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2021-04-02

Study results available
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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.

Conditions

  • Myasthenic Syndromes, Congenital

Interventions

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.

Sponsors & Collaborators

Principal Investigators

  • Thomas Crawford, M.D. · Johns Hopkins Pediatric Neurology

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
2 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-01-31
Primary Completion
2019-08-31
Completion
2019-10-31

Countries

  • United States

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02562066 on ClinicalTrials.gov