Ignite Creation Date:
2024-05-06 @ 1:15 AM
Last Modification Date:
2024-10-26 @ 11:01 AM
Study NCT ID:
NCT01765140
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2024-01-05
First Post:
2013-01-06
Brief Title:
Treatment Use of 34-Diaminopyridine
Sponsor:
Vern C Juel MD
Organization:
Duke University
Study Overview
Official Title:
Treatment Use of 34-Diaminopyridine in Congenital Myasthenic Syndrome
Status:
NO_LONGER_AVAILABLE
Status Verified Date:
2024-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This protocol has provided 34 diaminopyridine DAP under a treatment-use IND to patients with congenital myasthenic syndrome CMS It is currently closed to enrollment
Detailed Description:
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 turned 18 while in the program were re-consented as adults
The dose of DAP is determined individually for each patient Adults were started with a dose of 10 mg 3-4 times daily increased over several weeks to the dose that produced the maximum symptomatic response not to exceed 100 mg daily Pyridostigmine bromide PB was often 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 was 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 with 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 obtain DAP from commercial or other compassionate sources via a physician actively managing their care
CMS patients under age 18 years were included if their parent or guardian gave written permission Minors who turned 18 while in the program were re-consented as adults
The dose of DAP is determined individually for each patient Adults were started with a dose of 10 mg 3-4 times daily increased over several weeks to the dose that produced the maximum symptomatic response not to exceed 100 mg daily Pyridostigmine bromide PB was often 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 was 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 with 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 obtain DAP from commercial or other compassionate sources via a physician actively managing their care
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None