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

Descriptive Information
Brief Title † Treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) With 3, 4 DAP
Official Title † Treatment of Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes With 3, 4-Diaminopyridine
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.
Detailed Description
Study Phase N/A
Study Type † Expanded Access
Study Design † N/A
Primary Outcome Measure †
Secondary Outcome Measure †
Condition † Lambert Eaton Myasthenic Syndrome (LEMS)
Intervention † Drug3,4-diaminopyridine
Study Arms / Comparison Groups
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment †
Start Date † May 2005
Completion Date
Primary Completion Date May 2015
Eligibility 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
Gender Both
Ages 18 Years - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT01378546
Organization ID AAAB2528
Secondary IDs ††
Responsible Party Sponsor-Investigator
Study Sponsor † Louis H. Weimer, MD
Collaborators ††
Investigators † Principal Investigator: Louis H Weimer, MD, Columbia University
Information Provided By
Verification Date July 2013
First Received Date † June 20, 2011
Last Updated Date July 17, 2013
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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