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

Study Description

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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

Study Design

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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

Interventions

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Intervention Details:

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

Eligibility Criteria

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

Criteria

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

Contacts and Locations

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Locations

United States, New York
Columbia University Medical Center
New York, New York, United States, 10032

Sponsors and Collaborators

Louis H. Weimer, MD

Investigators

Principal Investigator:	Louis H Weimer, MD	Columbia University

More Information

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

Additional relevant MeSH terms:

Syndrome; Lambert-Eaton Myasthenic Syndrome; Disease; Pathologic Processes; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases	Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; 3,4-diaminopyridine; 4-Aminopyridine; Potassium Channel Blockers; Membrane Transport Modulators; Molecular Mechanisms of Pharmacological Action