Treatment Use of 3,4-Diaminopyridine

Myasthenic Syndromes, Congenital Clinical Trial

Official title:

Treatment Use of 3,4-Diaminopyridine in Congenital Myasthenic Syndrome

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT01765140
• Other study ID #: Pro00007811
• Status: No longer available

Study information

• Verified date: January 2024
• Source: Duke University
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial Summary

This protocol has provided 3,4 diaminopyridine (DAP) under a treatment-use IND to patients with congenital myasthenic syndrome (CMS). It is currently closed to enrollment.

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.

Recruitment information / eligibility

• Status: No longer available
• Enrollment: 0
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Diagnosis of 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

Related Conditions & MeSH terms

• Lambert-Eaton Myasthenic Syndrome
• Myasthenic Syndromes, Congenital
• Syndrome

Intervention

Drug:
• 3,4-diaminopyridine
Treatment use of 3,4-DAP for patients with congenital myasthenic syndrome (CMS)

Locations

Country	Name	City	State
United States	Duke University Hospital	Durham	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Vern C. Juel, M.D.

Country where clinical trial is conducted

United States, 

References & Publications (1)

Sanders DB, Juel VC, Harati Y, Smith AG, Peltier AC, Marburger T, Lou JS, Pascuzzi RM, Richman DP, Xie T, Demmel V, Jacobus LR, Ales KL, Jacobus DP; Dapper Study Team. 3,4-diaminopyridine base effectively treats the weakness of Lambert-Eaton myasthenia. M — View Citation