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

January 6, 2013
August 25, 2013
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Complete list of historical versions of study NCT01765140 on ClinicalTrials.gov Archive Site
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Compassionate Use of 3,4-Diaminopyridine
Compassionate 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) Investigational New Drug to patients with Lambert-Eaton myasthenic syndrome (LEMS) or congenital myasthenia gravis (CMG).

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.

Expanded Access
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  • Lambert Eaton Myasthenic Syndrome (LEMS)
  • Myasthenic Syndromes, Congenital
  • 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
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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 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
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
August 2013

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