Treatment Use of 3,4-Diaminopyridine

Expanded access is currently available for this treatment.
Verified June 2015 by Duke University
Information provided by (Responsible Party):
Vern C. Juel, M.D., Duke University Medical Center Identifier:
First received: January 6, 2013
Last updated: June 3, 2015
Last verified: June 2015
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).

Condition Intervention
Lambert Eaton Myasthenic Syndrome (LEMS)
Myasthenic Syndromes, Congenital
Drug: 3,4-diaminopyridine

Study Type: Expanded Access     What is Expanded Access?
Official Title: Treatment Use of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenia Gravis

Resource links provided by NLM:

Further study details as provided by Duke University:

Intervention Details:
    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
Detailed Description:

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.


Genders Eligible for Study:   Both

Inclusion Criteria:

  • Diagnosis of either Lambert Eaton myasthenic syndrome (LEMS) or congenital myasthenic syndrome (CMS)
  • Women of childbearing potential must have negative pregnancy test and agree to practice adequate contraception while taking DAP
  • Must be competent to give consent

Exclusion Criteria:

  • Known seizure disorder
  • Pregnancy
  • Known cardiac arrhythmia or evidence of significant arrhythmia on screening ECG
  • Known hepatic, renal or hematologic disease
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01765140

Contact: Vern C. Juel, M.D. 919-684-4044

United States, North Carolina
Duke University Hospital
Durham, North Carolina, United States, 27710
Contact: Vern C. Juel, M.D.    919-684-4044   
Principal Investigator: Vern C. Juel, M.D.         
Sponsors and Collaborators
Vern C. Juel, M.D.
Principal Investigator: Vern C. Juel, M.D. Duke University
  More Information

No publications provided

Responsible Party: Vern C. Juel, M.D., Associate Professor of Medicine, Duke University Medical Center Identifier: NCT01765140     History of Changes
Other Study ID Numbers: Pro00007811
Study First Received: January 6, 2013
Last Updated: June 3, 2015
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by Duke University:
3,4 diaminopyridine (DAP)

Additional relevant MeSH terms:
Myasthenic Syndromes, Congenital
Lambert-Eaton Myasthenic Syndrome
Autoimmune Diseases
Autoimmune Diseases of the Nervous System
Genetic Diseases, Inborn
Immune System Diseases
Neoplasms by Site
Nervous System Diseases
Nervous System Neoplasms
Neurodegenerative Diseases
Neuromuscular Diseases
Neuromuscular Junction Diseases
Paraneoplastic Syndromes
Paraneoplastic Syndromes, Nervous System
Pathologic Processes processed this record on November 25, 2015