Treatment Use of 3,4-Diaminopyridine

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01765140
Expanded Access Status : Available
First Posted : January 10, 2013
Last Update Posted : February 6, 2018
Information provided by (Responsible Party):
Vern C. Juel, M.D., Duke University

January 6, 2013
January 10, 2013
February 6, 2018
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 myasthenia (LEM) and congenital myasthenic syndrome (CMS).

The diagnosis of LEM 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 myasthenia (LEM)
    Other Name: DAP
  • Drug: 3,4-diaminopyridine
    Treatment use of 3,4-DAP for patients with congenital myasthenic syndrome (CMS)
    Other Name: DAP
Sanders DB, Juel VC, Harati Y, Smith AG, Peltier AC, Marburger T, Lou JS, Pascuzzi RM, Richman DP, Xie T, Demmel V, Jacobus LR, Aleš KL, Jacobus DP; Dapper Study Team. 3,4-diaminopyridine base effectively treats the weakness of Lambert-Eaton myasthenia. Muscle Nerve. 2018 Apr;57(4):561-568. doi: 10.1002/mus.26052. Epub 2018 Feb 2.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Contact: Missy Pittman, B.S.N. 919-681-5176
United States
Vern C. Juel, M.D., Duke University
Vern C. Juel, M.D.
Not Provided
Principal Investigator: Vern C. Juel, M.D. Duke University
Duke University
February 2018