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

Expanded access is no longer available for this treatment.
Information provided by (Responsible Party):
Louis H. Weimer, MD, Columbia University Identifier:
First received: June 20, 2011
Last updated: July 17, 2013
Last verified: July 2013

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 Intervention
Lambert Eaton Myasthenic Syndrome (LEMS) Drug: 3,4-diaminopyridine

Study Type: Expanded Access     What is Expanded Access?
Official Title: Treatment of Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes With 3, 4-Diaminopyridine

Resource links provided by NLM:

Further study details as provided by Louis H. Weimer, MD, Columbia University:

Study Start Date: May 2005
Estimated Primary Completion Date: May 2015 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: 3,4-diaminopyridine
    Treatment will begin with 5mg three times a day or less.

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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
  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 identifier: NCT01378546

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

Responsible Party: Louis H. Weimer, MD, Clinical Professor of Neurology, Columbia University Identifier: NCT01378546     History of Changes
Other Study ID Numbers: AAAB2528
Study First Received: June 20, 2011
Last Updated: July 17, 2013

Additional relevant MeSH terms:
Lambert-Eaton Myasthenic Syndrome
Paraneoplastic Syndromes, Nervous System
Paraneoplastic Syndromes
Pathologic Processes
Nervous System Neoplasms
Neoplasms by Site
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action processed this record on September 21, 2017