3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)

December 9, 2019 updated by: Oregon Health and Science University

3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenia

Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disorder which affects the nerve-muscle junction. The major symptoms of LEMS are progressive muscle weakness. Many patients experience other symptoms like dry mouth or impotence. Congenital Myasthenia (CM) is an inherited disorder with similar affects and symptoms.

3,4-Diaminopyridine (DAP) is an experimental drug that has improved strength in some subjects with (LEMS). There are no other accepted treatments for LEMS and DAP has relatively few side effects.

Study Overview

Status

No longer available

Conditions

Intervention / Treatment

Detailed Description

Subjects with clinically confirmed LEMS or CM will receive 3,4-diaminopyridine (3,4 DAP) by mouth in slowly increasing doses. Treatment will begin with 5-10 mg three times a day. A common final dosage is 15-20 mg four or five times a day, as clinically needed, and if tolerated. The upper limit is a total of 100 mg/day. Subjects will be monitored for strength and side effects via routine clinic visits at intervals of one month for the first three months, then every three months for the first year, and at least every six months thereafter. Treatment will be continued indefinitely if a good clinical response is achieved and side effects are tolerable.

Study Type

Expanded Access

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Diagnosis of LEMS or CM
  • If female and over the age of 9, must have a negative pregnancy test, and, if premenopausal, must be willing to practice an effective form of birth control.
  • Must be tested and found by ECG not to have a prolonged Q-Tc syndrome.
  • Must agree to have a second ECG at the time of peak drug effect.

Exclusion Criteria:

  • Known to have sensitivity to 3,4-DAP
  • History of clinical seizures or evidence of seizure activity on screening EEG
  • History of severe asthma

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Oregon Health and Science University

Collaborators

Jacobus Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

December 11, 2013

First Submitted That Met QC Criteria

December 11, 2013

First Posted (Estimate)

December 17, 2013

Study Record Updates

Last Update Posted (Actual)

December 11, 2019

Last Update Submitted That Met QC Criteria

December 9, 2019

Last Verified

December 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Jacobus compassionate program

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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