Treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) With 3, 4 DAP
| Status: | Available |
|---|---|
| Conditions: | Other Indications, Neurology |
| Therapuetic Areas: | Neurology, Other |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 7/16/2013 |
| Start Date: | May 2005 |
| Contact: | Louis H Weimer, MD |
| Email: | lhw1@columbia.edu |
| Phone: | 212-305-1330 |
Treatment of Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes With 3, 4-Diaminopyridine
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.
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
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