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

Expanded access is currently available for this treatment.
Verified April 2015 by Oregon Health and Science University
Jacobus Pharmaceutical
Information provided by (Responsible Party):
Tessa L Marburger, Oregon Health & Science University
ClinicalTrials.gov Identifier:
First received: December 11, 2013
Last updated: April 20, 2015
Last verified: April 2015

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.

Condition Intervention
Lambert-Eaton Myasthenic Syndrome (LEMS)
Congenital Myasthenia (CM)
Drug: 3,4-diaminopyridine

Study Type: Expanded Access     What is Expanded Access?
Official Title: 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenia

Resource links provided by NLM:

Further study details as provided by Oregon Health and Science University:

Intervention Details:
    Drug: 3,4-diaminopyridine
    10mg tablets for up to 100mg per day
    Other Name: 3,4DAP
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.

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02012933

Contact: Diana Dimitrova, PhD 503-494-7269 dimitrov@ohsu.edu

United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
Contact: Diana Dimitrova, PhD    503-494-7269    dimitrov@ohsu.edu   
Principal Investigator: Tessa Marburger, MD         
Sub-Investigator: Julie Khoury, MD         
Sponsors and Collaborators
Tessa L Marburger
Jacobus Pharmaceutical
  More Information

Responsible Party: Tessa L Marburger, Assistant Professor, Oregon Health & Science University
ClinicalTrials.gov Identifier: NCT02012933     History of Changes
Other Study ID Numbers: Jacobus compassionate program 
Study First Received: December 11, 2013
Last Updated: April 20, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Oregon Health and Science University:

Additional relevant MeSH terms:
Lambert-Eaton Myasthenic Syndrome
Myasthenic Syndromes, Congenital
Paraneoplastic Syndromes
Paraneoplastic Syndromes, Nervous System
Autoimmune Diseases
Autoimmune Diseases of the Nervous System
Genetic Diseases, Inborn
Immune System Diseases
Neoplasms by Site
Nervous System Diseases
Nervous System Neoplasms
Neurodegenerative Diseases
Neuromuscular Diseases
Neuromuscular Junction Diseases
Pathologic Processes
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Potassium Channel Blockers

ClinicalTrials.gov processed this record on May 26, 2016