Controlled Trial of 3,4-Diaminopyridine (3-4DAP) in Lambert-Eaton Myasthenic Syndrome (LEMS)
| Status: | Enrolling by invitation |
|---|---|
| Conditions: | Neurology, Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 45 - 65 |
| Updated: | 6/16/2018 |
| Start Date: | February 2004 |
| End Date: | December 2020 |
Controlled Trial of 3,4-Diaminopyridine in 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.
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.
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.
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.
Inclusion Criteria: -Male or female majority between 45 and 60 years of age
- diagnosed with Lambert-Eaton Myasthenic Syndrome.
- subjects must be taking full dose of pyridostigmine
Exclusion Criteria: - does subject have a history of liver problems?
- does subject have a history of prolonged QTc syndrome (which is a condition where
there is prolongation between the start of the Q wave and the end of the T wave in the
heart's electrical cycle).
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