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Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes
NCT02562066 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02562066
The ClinicalTrials.gov registry entry for NCT02562066 describes a study currently listed as completed. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 20 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Catalyst Pharmaceuticals, which has 6 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT02562066 reports 4 study locations spanning 4 distinct geographic areas — top geographies include California, Maryland, Massachusetts. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT02562066?
The interventions under investigation include: Placebo (DRUG), amifampridine phosphate (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT02562066?
This trial is sponsored by Catalyst Pharmaceuticals, which has 6 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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