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Clinical Trial Details — Status: Approved for marketing

Administrative data

NCT number NCT00872950
Other study ID # 2001-040
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information

Verified date March 2022
Source Lahey Clinic
Contact n/a
Is FDA regulated No
Health authority
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
Est. completion date
Est. primary completion date
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

Study Design


Related Conditions & MeSH terms


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, 

See also
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Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Completed NCT05408702 - Exercise in Autoimmune Myasthenia Gravis and Myasthenic Syndromes
Not yet recruiting NCT06436742 - A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS) Phase 1
Completed NCT01474980 - Pregnancy Outcomes in Congenital Myasthenie Syndrome N/A
Recruiting NCT06078553 - A Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes (CMS)
No longer available NCT02189720 - Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
No longer available NCT03062631 - Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia