Clinical Trials Logo

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).


Clinical Trial 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT00872950
Study type Expanded Access
Source Lahey Clinic
Contact
Status Approved for marketing
Phase

See also
  Status Clinical Trial Phase
Completed NCT01203592 - Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes Phase 1
Recruiting NCT01403402 - Congenital Muscle Disease Study of Patient and Family Reported Medical Information
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 NCT03062631 - Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia
No longer available NCT02189720 - Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome