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

Descriptive Information
Brief Title † 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)
Official Title † 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenia
Brief 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.
Detailed Description Subjects with clinically confirmed LEMS or CM will receive 3,4-diaminopyridine (3,4 DAP) by mouth in slowly increasing doses. Treatment will begin with 5-10 mg three times a day. A common final dosage is 15-20 mg four or five times a day, as clinically needed, and if tolerated. The upper limit is a total of 100 mg/day. Subjects will be monitored for strength and side effects via routine clinic visits at intervals of one month for the first three months, then every three months for the first year, and at least every six months thereafter. Treatment will be continued indefinitely if a good clinical response is achieved and side effects are tolerable.
Study Phase N/A
Study Type † Expanded Access
Study Design † N/A
Primary Outcome Measure †
Secondary Outcome Measure †
Condition † Lambert-Eaton Myasthenic Syndrome (LEMS) Congenital Myasthenia (CM)
Intervention † Drug3,4-diaminopyridine
Study Arms / Comparison Groups
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment †
Start Date †
Completion Date
Primary Completion Date
Eligibility Criteria † 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
Gender Both
Ages 2 Years - N/A
Accepts Healthy Volunteers
Contacts †† Diana Dimitrova, PhD, 503-494-7269, dimitrov@ohsu.edu
Location Countries † United States
Administrative Information
NCT ID † NCT02012933
Organization ID Jacobus compassionate program
Secondary IDs ††
Responsible Party Sponsor-Investigator
Study Sponsor † Tessa L Marburger
Collaborators †† Jacobus Pharmaceutical
Investigators † : ,
Information Provided By
Verification Date April 2015
First Received Date † December 11, 2013
Last Updated Date April 20, 2015
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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