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NCT02562066 · ClinicalTrials.gov registry record · Phase 3
Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes
A Phase 3 study of Myasthenic Syndromes, Congenital, sponsored by Catalyst Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 20
- Enrollment target
- 4
- Study locations
NCT02562066: Completed Phase 3 study of Myasthenic Syndromes, Congenital, sponsored by Catalyst Pharmaceuticals.
NCT02562066 is a Phase 3 study of Myasthenic Syndromes, Congenital that has completed, run by Catalyst Pharmaceuticals. The registered enrollment target is 20 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower). The trial reports 4 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02562066, a Phase 3 study of Myasthenic Syndromes, Congenital, has completed, sponsored by Catalyst Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 20 participants
- Enrollment target
- 4
- Study locations
Study 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.
Primary Outcome
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 to D8), Study Period 2 (difference in SGI score from D21 to D29) and the change in CFB from Study P
Conditions Studied
Interventions
- DRUG Placebo
- DRUG amifampridine phosphate
Study Locations (4)
California
- UCLA Department of Neurology - Los Angeles
Maryland
- Johns Hopkins Pediatric Neurology - Baltimore
Massachusetts
- Boston Children's Hospital - Boston
Ohio
- Ohio State University - Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2016-01 |
| Est. Completion | 2019-10 |
| Phase | Phase 3 |
What the finished NCT02562066 record still lists
NCT02562066 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 20 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower).
The record links to 1 condition, with Myasthenic Syndromes, Congenital appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT02562066 reports a single indexed study location in California, Maryland, Massachusetts.
Frequently Asked Questions
What is clinical trial NCT02562066 about?
NCT02562066 is a clinical study titled "Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes". 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 phosphat...
What is the current status of trial NCT02562066?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 20 participants. The study started on 2016-01. Estimated completion is 2019-10.
What conditions does trial NCT02562066 study?
This clinical trial studies the following conditions: Myasthenic Syndromes, Congenital.
What interventions are being tested in trial NCT02562066?
The interventions under investigation include: Placebo (DRUG), amifampridine phosphate (DRUG).
Who is sponsoring clinical trial NCT02562066?
This trial is sponsored by Catalyst Pharmaceuticals, which has 10 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02562066 being conducted?
This trial has 4 study locations across California, Maryland, Massachusetts, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT02562066's enrollment target sits among peer trials
20 1956th of 2000 higher than 32 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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