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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. Read our disclaimer for details. Identifier: NCT01765140
Expanded Access Status : No longer available
First Posted : January 10, 2013
Last Update Posted : January 26, 2021
Information provided by (Responsible Party):
Vern C. Juel, M.D., Duke University

Brief Summary:
This protocol has provided 3,4 diaminopyridine (DAP) under a treatment-use IND to patients with congenital myasthenic syndrome (CMS). It is currently closed to enrollment.

Condition or disease Intervention/treatment
Myasthenic Syndromes, Congenital Drug: 3,4-diaminopyridine

Detailed Description:

CMS diagnoses are made based on clinical, electromyographic and molecular genetic findings, and all patients have been referred to the PI for DAP treatment. This study enrolled minors and adults.

CMS patients under age 18 years were included if their parent or guardian gave written permission. Minors who turn 18 while in the program will be re-consented as adults.

The dose of DAP is determined individually for each patient. Adults are started with a dose of 10 mg 3-4 times daily, increased 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 and increased to the dose that produced the best response, not to exceed 360 mg daily. In children, equivalent doses of these medications is calculated on a surface area basis. The doses of DAP and PB are 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.

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Study Type : Expanded Access
Official Title: Treatment Use of 3,4-Diaminopyridine in Congenital Myasthenic Syndrome

Intervention Details:
  • Drug: 3,4-diaminopyridine
    Treatment use of 3,4-DAP for patients with congenital myasthenic syndrome (CMS)
    Other Name: DAP

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All

Inclusion Criteria:

  • Diagnosis of 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01765140

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United States, North Carolina
Duke University Hospital
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
Vern C. Juel, M.D.
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Principal Investigator: Vern C. Juel, M.D. Duke University
Publications of Results:
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Responsible Party: Vern C. Juel, M.D., Professor of Neurology, Duke University Identifier: NCT01765140    
Other Study ID Numbers: Pro00007811
First Posted: January 10, 2013    Key Record Dates
Last Update Posted: January 26, 2021
Last Verified: January 2021
Keywords provided by Vern C. Juel, M.D., Duke University:
3,4 diaminopyridine (DAP)
Additional relevant MeSH terms:
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Lambert-Eaton Myasthenic Syndrome
Myasthenic Syndromes, Congenital
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Genetic Diseases, Inborn
Neuromuscular Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action