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Treatment Use of 3,4-Diaminopyridine

Expanded access is currently available for this treatment.
Verified April 2014 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: April 27, 2014
Last verified: April 2014

January 6, 2013
April 27, 2014
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Complete list of historical versions of study NCT01765140 on ClinicalTrials.gov Archive Site
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Treatment Use of 3,4-Diaminopyridine
Treatment Use of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenia Gravis

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
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  • Lambert Eaton Myasthenic Syndrome (LEMS)
  • Myasthenic Syndromes, Congenital
  • 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
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
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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
Both
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Contact: Vern C. Juel, M.D. 919-684-4044 vern.juel@duke.edu
United States
 
NCT01765140
Pro00007811
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Vern C. Juel, M.D., Duke University Medical Center
Vern C. Juel, M.D.
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Principal Investigator: Vern C. Juel, M.D. Duke University School of Medicine
Duke University
April 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP