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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02012933
Expanded Access Status : Available
First Posted : December 17, 2013
Last Update Posted : April 21, 2015
Jacobus Pharmaceutical
Information provided by (Responsible Party):
Tessa L Marburger, Oregon Health and Science University

Brief Summary:

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 or disease Intervention/treatment
Lambert-Eaton Myasthenic Syndrome (LEMS) Congenital Myasthenia (CM) Drug: 3,4-diaminopyridine

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.

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

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

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02012933

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Contact: Diana Dimitrova, PhD 503-494-7269

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

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Responsible Party: Tessa L Marburger, Assistant Professor, Oregon Health and Science University Identifier: NCT02012933     History of Changes
Other Study ID Numbers: Jacobus compassionate program
First Posted: December 17, 2013    Key Record Dates
Last Update Posted: April 21, 2015
Last Verified: April 2015
Keywords provided by Tessa L Marburger, Oregon Health and Science University:
Additional relevant MeSH terms:
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Lambert-Eaton Myasthenic Syndrome
Muscle Weakness
Myasthenic Syndromes, Congenital
Pathologic Processes
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Genetic Diseases, Inborn
Neuromuscular Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Potassium Channel Blockers
Membrane Transport Modulators