3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)

Congenital Myasthenic Syndrome Clinical Trial

— LEMS  

Official title:

Open Label Trial Of 3,4-Diaminopyridine In Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS)

Clinical Trial Details — Status: Approved for marketing

Administrative data

• NCT number: NCT00872950
• Other study ID #: 2001-040
• Status: Approved for marketing

Study information

• Verified date: March 2022
• Source: Lahey Clinic
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial Summary

The purpose of this study is to determine the effectiveness and adverse effects of 3,4-diaminopyridine for the treatment of the Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS).

Description:

This is an open-label, non-randomized, non-comparative expanded access study. Up to 25 patients with clinically proven paraneoplastic or primary autoimmune LEMS per EMG and positive voltage-gated calcium channel antibody serology, OR patients with clinically proven CMS per electromyogram (EMG), biopsy or genetic testing who meet the selection criteria outlined in sections 3.1 and 3.2 will be enrolled in this study. Subjects will receive 3,4-diaminopyridine (3,4-DAP) starting with a low dose and titrating up per efficacy and patient tolerance to a maximum daily dose of 100mg . Treatment will continue until the subject experiences a treatment-limiting toxicity, voluntarily withdraws consent, transfers to another site, dies, becomes lost to follow-up, is no longer receiving clinical benefit from 3,4-DAP (in the opinion of the subject and/or the investigator), or the Jacobus Pharmaceuticals or the FDA discontinues the study.

Recruitment information / eligibility

• Status: Approved for marketing
• Enrollment: 0
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• LEMS diagnosis OR Pre or Post Synaptic CMS diagnosis

• 18 years or older

• Females must have negative pregnancy test and be willing to practice an effective form of birth control

• No prolonged QT syndrome as indicated by baseline EKG

Exclusion Criteria:

• Known sensitivity to 3,4-DIAMINOPYRIDINE

• History of seizures and/or severe asthma

Related Conditions & MeSH terms

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

Intervention

Drug:
• 3,4-DIAMINOPYRIDINE
Treatment will begin with a low dose and will be increased as clinically needed as tolerated. The upper limit will be a total of 100mg/day.

Locations

Country	Name	City	State
United States	Lahey Hospital & Medical Center	Burlington	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Lahey Clinic

Country where clinical trial is conducted

United States, 

External Links

•   Lahey Hospital & Medical Center