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

Lambert Eaton Myasthenic Syndrome (LEMS) Clinical Trial

Official title:

Treatment of Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes With 3, 4-Diaminopyridine

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT01378546
• Other study ID #: AAAB2528
• Status: No longer available
• Phase: N/A
• First received: June 20, 2011
• Last updated: July 17, 2013
• Start date: May 2005

Study information

• Verified date: July 2013
• Source: Columbia University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Expanded Access

Clinical Trial 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.

Recruitment information / eligibility

• Status: No longer available
• Enrollment: 0
• Est. primary completion date: May 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

N/A

Related Conditions & MeSH terms

• Lambert Eaton Myasthenic Syndrome (LEMS)
• Lambert-Eaton Myasthenic Syndrome
• Myasthenic Syndromes, Congenital
• Syndrome

Intervention

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

Locations

Country	Name	City	State
United States	Columbia University Medical Center	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Louis H. Weimer, MD

Country where clinical trial is conducted

United States,