Who is sponsoring NCT02562066?

NCT02562066 is sponsored by Catalyst Pharmaceuticals, Inc..

What drugs are being tested in NCT02562066?

Interventions in NCT02562066: amifampridine phosphate, Placebo.

What condition does NCT02562066 study?

NCT02562066 studies Myasthenic Syndromes, Congenital.

Is NCT02562066 still recruiting?

NCT02562066 is currently completed. Last updated 8 March 2021.

Are results published for NCT02562066?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2021-03-08 · How we verify

NCT02562066

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

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)

Completed Phase 3 Results posted Last updated 8 March 2021

What this trial tests

Phase 3 trial testing amifampridine phosphate in Myasthenic Syndromes, Congenital in 20 participants. Completed in 1 October 2019.

Timeline

1 January 2016

Primary endpoint
1 August 2019

1 October 2019

Quick facts

Lead sponsor	Catalyst Pharmaceuticals, Inc.
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	quadruple
Primary purpose	treatment
Enrollment	20
Start date	1 January 2016
Primary completion	1 August 2019
Estimated completion	1 October 2019
Sites	4 locations across United States

Drugs / interventions tested

amifampridine phosphate — full drug profile →
Placebo

Conditions studied

Myasthenic Syndromes, Congenital — all drugs for Myasthenic Syndromes, Congenital →

Sponsor

Catalyst Pharmaceuticals, Inc. — full company profile →

Who can join

Adults 2 to 70, any sex, with Myasthenic Syndromes, Congenital. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis
Time frame: Study Period 1: Baseline (Day 0), Day 8; Study Period 2: Baseline (Day 21), Day 29
Subject Global Impression (SGI) rates the subject's impression of the effects of the study medication during the preceding week with max score =7 (most satisfied) and min score =1 (least satisfied). It will be completed at D0 (baseline for Study Period 1), D8, D21 (baseline for Study Period 2), and D29. Change from baseline (CFB) will be assessed for Study Period 1 (difference in SGI score from D0

Sponsor's own description

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.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

Treating pediatric neuromuscular disorders: The future is now.
Dowling JJ, D Gonorazky H, Cohn RD, Campbell C. · · 2018 · cited 83× · PMID 28889642 · DOI 10.1002/ajmg.a.38418
Aminopyridines for the treatment of neurologic disorders.
Strupp M, Teufel J, Zwergal A, Schniepp R, et al · · 2017 · cited 46× · PMID 28243504 · DOI 10.1212/cpj.0000000000000321

Verify or expand the search:

Other recruiting trials for Myasthenic Syndromes, Congenital

Currently open trials in the same condition.

NCT06630650 — A Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes · recruiting

Other Catalyst Pharmaceuticals, Inc. trials

Trials by the same sponsor.

NCT03819660 — Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 · Phase 2 · terminated
NCT03781479 — Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients · Phase 2 · completed
NCT03579966 — Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis) · Phase 3 · terminated
NCT03304054 — Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG · Phase 3 · completed
NCT02189720 — Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome · approved for marketing

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT02562066 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Catalyst Pharmaceuticals, Inc.
Last refreshed: 8 March 2021

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02562066.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing