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Treatment Use of 3,4-Diaminopyridine

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ClinicalTrials.gov Identifier: NCT01765140
Recruitment Status : Available
First Posted : January 10, 2013
Last Update Posted : January 25, 2017
Information provided by (Responsible Party):

January 6, 2013
January 10, 2013
January 25, 2017
Treatment Use of 3,4-Diaminopyridine
This is a continuing project to provide 3,4 diaminopyridine (DAP) under a treatment-use IND to patients with Lambert-Eaton myasthenic syndrome (LEMS) and congenital myasthenic syndrome (CMS).

The diagnosis of LEMS or CMS 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.

CMS patients under age 18 years will be included if their parent or guardian gives written permission. Minors who turn 18 while in the program 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 3-4 times daily, increasing over several weeks to the dose that produces the maximum symptomatic response, not to exceed 100 mg daily. Pyridostigmine bromide (PB) may be added at low doses, increasing to the dose that produces the best response, not to exceed 360 mg daily. In children, equivalent doses of these medications will be given calculated on a surface area basis. The doses of DAP and PB 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 evaluations 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.

Expanded Access
  • Drug: 3,4-diaminopyridine
    Treatment use of 3,4-DAP for patients with Lambert Eaton myasthenic syndrome (LEMS)
    Other Name: DAP
  • Drug: 3,4-diaminopyridine
    Treatment use of 3,4-DAP for patients with congenital myasthenic syndrome (CMS)
    Other Name: DAP
Not Provided
Contact: Missy Pittman, B.S.N. 919-681-5176 melissa.pittman@duke.edu
United States
Vern C. Juel, M.D., Duke University Medical Center
Vern C. Juel, M.D.
Not Provided
Principal Investigator: Vern C. Juel, M.D. Duke University
Duke University
January 2017