NO_LONGER_AVAILABLENot Applicable

3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)

United States

Plain-language summary

Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disorder which affects the nerve-muscle junction. The major symptoms of LEMS are progressive muscle weakness. Many patients experience other symptoms like dry mouth or impotence. Congenital Myasthenia (CM) is an inherited disorder with similar affects and symptoms. 3,4-Diaminopyridine (DAP) is an experimental drug that has improved strength in some subjects with (LEMS). There are no other accepted treatments for LEMS and DAP has relatively few side effects.

Who can participate

Age range2 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Diagnosis of LEMS or CM * If female and over the age of 9, must have a negative pregnancy test, and, if premenopausal, must be willing to practice an effective form of birth control. * Must be tested and found by ECG not to have a prolonged Q-Tc syndrome. * Must agree to have a second ECG at the time of peak drug effect. Exclusion Criteria: * Known to have sensitivity to 3,4-DAP * History of clinical seizures or evidence of seizure activity on screening EEG * History of severe asthma

Trial details

NCT IDNCT02012933

SponsorOregon Health and Science University

Sponsor typeOTHER

Study typeEXPANDED_ACCESS

View on ClinicalTrials.gov More Lambert-Eaton Myasthenic Syndrome (LEMS) trials