Ignite Creation Date:
2025-12-25 @ 2:47 AM
Ignite Modification Date:
2026-04-06 @ 3:23 PM
Study NCT ID:
NCT02012933
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2019-12-11
First Post:
2013-12-11
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)
Sponsor:
Oregon Health and Science University
Organization:
Study Overview
Official Title:
3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenia
Status:
NO_LONGER_AVAILABLE
Status Verified Date:
2019-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
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 Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: