Study on 3,4-Diaminopyridine for Treating Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes

Sponsor: Lahey Clinic

Expanded Access
Approved for Marketing
Sponsor score: 40
Inactive

ClinicalTrials ID:NCT00872950

Provider View

This clinical trial is exploring the use of 3,4-diaminopyridine to treat Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS), focusing on its effectiveness and potential side effects.

Patient Parameters

Program Overview

The aim of this study is to find out how well 3,4-diaminopyridine works and what side effects it might have for people with Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS).

Description

This study is open to up to 25 patients who have been diagnosed with either Lambert-Eaton Myasthenic Syndrome (LEMS) or Congenital Myasthenic Syndromes (CMS) through specific medical tests. Participants will take 3,4-diaminopyridine (3,4-DAP), starting with a low dose that can be increased based on how well it works and how well the patient tolerates it, up to a maximum of 100mg per day. The treatment will continue until the patient experiences significant side effects, chooses to stop, moves to another location, passes away, cannot be contacted, no longer benefits from the treatment, or if the study is stopped by the pharmaceutical company or the FDA.

Eligibility Criteria

Inclusion Criteria

  • Diagnosed with Lambert-Eaton Myasthenic Syndrome (LEMS) or Congenital Myasthenic Syndromes (CMS)
  • 18 years or older
  • Women must have a negative pregnancy test and agree to use effective birth control
  • No prolonged QT syndrome as shown by an EKG test

Exclusion Criteria

  • Known allergy to 3,4-diaminopyridine
  • History of seizures or severe asthma

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