Clinical Trial: Controlled Trial of 3,4-Diaminopyridine (3-4DAP) in Lambert-Eaton Myasthenic Syndrome (LEMS)
Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional
Official Title: Controlled Trial of 3,4-Diaminopyridine in LEMS
Brief Summary: 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.
Detailed Summary:
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.
Sponsor: Dartmouth-Hitchcock Medical Center
Current Primary Outcome: Change in muscle weakness [ Time Frame: Month 1, Month 2, Month 3, then changing to every 6 months once patient is stable ]
Original Primary Outcome: Change in muscle weakness [ Time Frame: Month 1, Month 2, Month 3, then changing to every 6 months once patient is stable ]
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Dartmouth-Hitchcock Medical Center
Dates:
Date Received: March 14, 2014
Date Started: February 2004
Date Completion:
Last Updated: August 1, 2016
Last Verified: August 2016
