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

NCT01378546 | Unknown

• 1 other identifier: AAAB2528
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Lambert Eaton Myasthenic Syndrome (LEMS)

Interventions

3,4-diaminopyridine DRUG

Treatment will begin with 5mg three times a day or less.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Louis H. Weimer, MD: lead

Study Sites (1)

Columbia University Medical Center

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

Lambert-Eaton Myasthenic Syndrome

Interventions

Amifampridine

Condition Hierarchy (Ancestors)

Myasthenia Gravis; 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

Intervention Hierarchy (Ancestors)

4-Aminopyridine; Aminopyridines; Amines; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Louis H Weimer, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Clinical Professor of Neurology

Study Record Dates

• First Submitted: June 20, 2011
• First Posted: June 22, 2011
• Study Start: May 1, 2005
• Primary Completion: May 1, 2015
• Last Updated: July 18, 2013

Record last verified: 2013-07

Locations

US (1)