Congenital Myasthenia (CM) Clinical Trial
Official title:
3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenia
NCT number | NCT02012933 |
Other study ID # | Jacobus compassionate program |
Secondary ID | |
Status | No longer available |
Phase | |
First received | |
Last updated |
Verified date | December 2019 |
Source | Oregon Health and Science University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Expanded Access |
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.
Status | No longer available |
Enrollment | 0 |
Est. completion date | |
Est. primary completion date | |
Accepts healthy volunteers | |
Gender | All |
Age group | 2 Years and older |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
United States | Oregon Health & Science University | Portland | Oregon |
Lead Sponsor | Collaborator |
---|---|
Oregon Health and Science University | Jacobus Pharmaceutical |
United States,