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Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome

NCT02189720 · Status: APPROVED_FOR_MARKETING · Type: EXPANDED_ACCESS

Last updated 2025-03-26

No results posted yet for this study

Summary

Primary:

The primary objective of this study under the original protocol was to provide neuromuscular specialists and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus until the product became commercially available.

Secondary:

The secondary objective of this study under the original protocol was to provide additional long-term safety data on amifampridine phosphate in patients.

Primary The primary objective of this study after its fifth amendment was to provide access to amifampridine phosphate therapy to pediatric patients with LEMS, and pediatric and adult patients with CMS until the product became commercially available for these indications or development of the product for the indication was terminated.

Secondary:

The secondary objective of this study after its fifth amendment was to assess the long-term safety of amifampridine phosphate in pediatric patients with LEMS, and pediatric and adult patients with CMS.

Conditions

  • Congenital Myasthenic Syndrome
  • Lambert-Eaton Myasthenic Syndrome
  • Nystagmus, Acquired

Interventions

DRUG

Amifampridine Phosphate

Dosage form: tablets containing the equivalent of 10 mg amifampridine per tablet. Amifampridine Phosphate given based on Investigator assessment of optimal neuromuscular benefit. In adult patients \> 16 years of age, Doses ranged from 30 mg to 80 mg, divided into doses taken 3 to 4 times per day and in pediatric patients age \< 16 years the maximum daily dose was 60 mg/day. No single dose to exceed 20 mg.

Sponsors & Collaborators

Principal Investigators

  • Perry Shieh, MD, PhD · University of California, Los Angeles

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

More Related Trials

Entities

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 NCT02189720 on ClinicalTrials.gov