Trial Information

Compassionate Use of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenia Gravis

The diagnosis of LEMS or CMG will have been made based on clinical and electromyographic
findings, and all patients will have been referred to the PI for DAP treatment. This study
will enroll minors and adults.

CMG patients under age 18 will be included if their parent or guardian gives written
permission. Minors who turn 18 while on the study will be re-consented as adults.

The dose of DAP will be determined individually for each patient. Adults will start with a
dose of 10 mg 3 or 4 times a day, increasing over several weeks to the dose that produces
the maximum symptomatic response, not to exceed 100 mg/day. Mestinon will then be added at
low doses, increasing to the dose that produces the best response, not to exceed 360 mg/day.
In children, equivalent doses of these medications will be given calculated on a surface
area basis. The doses of DAP and Mestinon will be periodically adjusted to assure that the
smallest effective doses are used.

Patients who achieve significant clinical benefit from DAP, as judged by the study PI and
the patient, may continue taking DAP as long as the drug is available from the sponsor, and
as long as they return for regular follow-up at the Duke MG Clinic. Patients who are unable
to return for regular follow-up will be required to have their local physician obtain DAP
for them from the sponsor.