Controlled Trial of 3,4-Diaminopyridine (3-4DAP) in Lambert-Eaton Myasthenic Syndrome (LEMS)
The main purpose for this study is to provide access to 3,4 DAP, a drug which has demonstrated to be effective in treating weakness associated with Lambert-Eaton Myasthenic Syndrome. LEMS is a rare autoimmune cause of a defect in neuromuscular transmission. The disorder is clinically characterized by fluctuating muscle weakness, hyporeflexia and autonomic dysfunction.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Controlled Trial of 3,4-Diaminopyridine in LEMS|
- Change in muscle weakness [ Time Frame: Month 1, Month 2, Month 3, then changing to every 6 months once patient is stable ] [ Designated as safety issue: Yes ]Muscle weakness will be assessed monthly for the first 3 months based on office visits and blood tests (complete blood count (CBC), electrolytes, glucose, liver function tests, blood urea nitrogen (BUN), creatinine). Muscle weakness will then be assessed every 6 months once the patient is stabilized based on office visits and blood tests (CBC, electrolytes, glucose, liver function tests, BUN, creatinine).
|Study Start Date:||February 2004|
|Estimated Primary Completion Date:||December 2020 (Final data collection date for primary outcome measure)|
|Experimental: 3-4 Diaminopyridine (DAP)||
Drug: 3-4 Diaminopyridine
Other Name: 3-4 DAP
More than half of LEMS cases are associated with malignancy, usually small cell lung cancer. These paraneoplastic cases progress more quickly than primary autoimmune LEMS. An overlap syndrome with other autoimmune diseases is often detected in LEMS patients.
3,4 DAP is effective in LEMS because it increases calcium influx into the nerve terminal by blocking potassium efflux and thereby prolonging the presynaptic action potential. 3,4 DAP is less likely to provoke epileptic seizures than its precursor, 4-aminopyridine, because it is less able to cross the blood-brain barrier. 3,4 DAP is effective in increasing strength and improving autonomic symptoms in LEMS patients of both the primary autoimmune and paraneoplastic etiologies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02090725
|United States, New Hampshire|
|Dartmouth-Hitchcock Medical Center|
|Lebanon, New Hampshire, United States, 03221|
|Principal Investigator:||Jeffrey A. Cohen, MD||Dartmouth-Hitchcock Medical Center|