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Trial record 11 of 12 for:    Congenital Myasthenia

Treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) With 3, 4 DAP

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. Read our disclaimer for details. Identifier: NCT01378546
Expanded Access Status : No longer available
First Posted : June 22, 2011
Last Update Posted : July 18, 2013
Information provided by (Responsible Party):
Louis H. Weimer, MD, Columbia University

Brief Summary:

Lambert Eaton Myasthenic Syndrome (LEMS) is rare neurological disorder that results in muscle weakness and limited reflex activity. More than half of LEMS cases are associated with a malignancy, usually small cell lung cancer, and tend to progress more quickly than cases not coupled with malignant cells.

3,4diaminopyridine (3,4DAP)is a drug that has been demonstrated to be effective in treating the weakness associated with LEMS as it increases strength and improves autonomic symptoms in LEMS patients. It is not currently approved by the FDA for use in the United States. The investigators plan to use 3,4DAP to treat patients with LEMS here at the Columbia University MDA/ALS Research Center.

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

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Study Type : Expanded Access
Official Title: Treatment of Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes With 3, 4-Diaminopyridine
Study Start Date : May 2005
Estimated Primary Completion Date : May 2015

Intervention Details:
  • Drug: 3,4-diaminopyridine
    Treatment will begin with 5mg three times a day or less.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Be diagnosed with LEMS or a type of CMS likely to respond to 3, 4-DAP.
  • If female of childbearing age, have negative pregnancy test, and be willing to practice and effective form of birth control during the study.
  • Tested and found by ECG not to have a prolonged QTc syndrome.
  • Agrees to have a second ECG at the time of peak drug effect. Has understood and signed the Informed Consent.

Exclusion Criteria:

  • Is known to have a sensitivity to 3, 4-DAP.
  • Has a history of past or current seizures or of severe asthma, or has an epileptiform EEG.
  • Is believed by the investigator to be unable to comply with the protocol.
  • Is unable to give informed consent.
  • No patient will be excluded based on race, ethnicity, gender, or HIV status

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): NCT01378546

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United States, New York
Columbia University Medical Center
New York, New York, United States, 10032
Sponsors and Collaborators
Louis H. Weimer, MD
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Principal Investigator: Louis H Weimer, MD Columbia University

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Responsible Party: Louis H. Weimer, MD, Clinical Professor of Neurology, Columbia University Identifier: NCT01378546     History of Changes
Other Study ID Numbers: AAAB2528
First Posted: June 22, 2011    Key Record Dates
Last Update Posted: July 18, 2013
Last Verified: July 2013

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