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Compassionate Use of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenia Gravis


N/A
N/A
N/A
Open (Enrolling)
Both
Lambert Eaton Myasthenic Syndrome (LEMS), Myasthenic Syndromes, Congenital

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Trial Information

Compassionate Use of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenia Gravis


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.


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

Type of Study:

Expanded Access

Study Design:

N/A

Principal Investigator

Vern C. Juel, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

Duke University School of Medicine

Authority:

United States: Food and Drug Administration

Study ID:

Pro00007811

NCT ID:

NCT01765140

Start Date:

Completion Date:

Related Keywords:

  • Lambert Eaton Myasthenic Syndrome (LEMS)
  • Myasthenic Syndromes, Congenital
  • 3,4 diaminopyridine (DAP)
  • Lambert-Eaton Myasthenic Syndrome
  • Myasthenic Syndromes, Congenital

Name

Location

Duke University HospitalDurham, North Carolina  27710