Compassionate Use of 3,4-Diaminopyridine

Expanded access is currently available for this treatment.
Verified August 2013 by Duke University
Sponsor:
Information provided by (Responsible Party):
Vern C. Juel, M.D., Duke University Medical Center
ClinicalTrials.gov Identifier:
NCT01765140
First received: January 6, 2013
Last updated: August 25, 2013
Last verified: August 2013
  Purpose

This is a continuing project to provide 3,4 diaminopyridine (DAP) under a treatment-use (IND) Investigational New Drug to patients with Lambert-Eaton myasthenic syndrome (LEMS) or congenital myasthenia gravis (CMG).


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

Study Type: Expanded Access     What is Expanded Access?
Official Title: Compassionate 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
    Compassionate use of 3,4-DAP for patients with Lambert Eaton myasthenic syndrome (LEMS)
    Other Name: DAP
    Drug: 3,4-diaminopyridine
    Compassionate use of 3,4-DAP for patients with congenital myasthenic gravis (CMG)
    Other Name: DAP
Detailed Description:

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.

  Eligibility

Genders Eligible for Study:   Both
Criteria

Inclusion Criteria:

  • diagnosis of either Lambert Eaton myasthenic syndrome (LEMS) or congenital myasthenic gravis (CMG)
  • 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 these on screening ECG
  • known hepatic, renal or hematologic disease
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01765140

Contacts
Contact: Vern C. Juel, M.D. 919-684-4044 vern.juel@duke.edu

Locations
United States, North Carolina
Duke University Hospital
Durham, North Carolina, United States, 27710
Contact: Vern C. Juel, M.D.    919-684-4044    vern.juel@duke.edu   
Principal Investigator: Vern C. Juel, M.D.         
Sponsors and Collaborators
Vern C. Juel, M.D.
Investigators
Principal Investigator: Vern C. Juel, M.D. Duke University School of Medicine
  More Information

No publications provided

Responsible Party: Vern C. Juel, M.D., Associate Professor of Medicine, Duke University Medical Center
ClinicalTrials.gov Identifier: NCT01765140     History of Changes
Other Study ID Numbers: Pro00007811
Study First Received: January 6, 2013
Last Updated: August 25, 2013
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:
Lambert-Eaton Myasthenic Syndrome
Myasthenic Syndromes, Congenital
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Neoplasms
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
3,4-diaminopyridine
4-Aminopyridine
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Cardiovascular Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on April 23, 2014