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.|
|ClinicalTrials.gov Identifier: NCT01765140|
Expanded Access Status : No longer available
First Posted : January 10, 2013
Last Update Posted : January 26, 2021
|Condition or disease||Intervention/treatment|
|Myasthenic Syndromes, Congenital||Drug: 3,4-diaminopyridine|
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.
|Study Type :||Expanded Access|
|Official Title:||Treatment Use of 3,4-Diaminopyridine in Congenital Myasthenic Syndrome|
- Drug: 3,4-diaminopyridine
Treatment use of 3,4-DAP for patients with congenital myasthenic syndrome (CMS)Other Name: DAP
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 ClinicalTrials.gov identifier (NCT number): NCT01765140
|United States, North Carolina|
|Duke University Hospital|
|Durham, North Carolina, United States, 27710|
|Principal Investigator:||Vern C. Juel, M.D.||Duke University|