3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)

NCT02012933 | Unknown

• 1 other identifier: Jacobus compassionate program
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disorder which affects the nerve-muscle junction. The major symptoms of LEMS are progressive muscle weakness. Many patients experience other symptoms like dry mouth or impotence. Congenital Myasthenia (CM) is an inherited disorder with similar affects and symptoms. 3,4-Diaminopyridine (DAP) is an experimental drug that has improved strength in some subjects with (LEMS). There are no other accepted treatments for LEMS and DAP has relatively few side effects.

Conditions

Lambert-Eaton Myasthenic Syndrome (LEMS); Congenital Myasthenia (CM)

Keywords

3,4DAP, LEMS, CM

Interventions

3,4-diaminopyridine DRUG

10mg tablets for up to 100mg per day

Also known as: 3,4DAP

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of LEMS or CM
• If female and over the age of 9, must have a negative pregnancy test, and, if premenopausal, must be willing to practice an effective form of birth control.
• Must be tested and found by ECG not to have a prolonged Q-Tc syndrome.
• Must agree to have a second ECG at the time of peak drug effect.

You may not qualify if:

• Known to have sensitivity to 3,4-DAP
• History of clinical seizures or evidence of seizure activity on screening EEG
• History of severe asthma

Sponsors & Collaborators

• Oregon Health and Science University: lead
• Jacobus Pharmaceutical: collaborator

Study Sites (1)

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

MeSH Terms

Conditions

Lambert-Eaton Myasthenic Syndrome; Myasthenic Syndromes, Congenital

Interventions

Amifampridine

Condition Hierarchy (Ancestors)

Myasthenia Gravis; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

4-Aminopyridine; Aminopyridines; Amines; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 11, 2013
• First Posted: December 17, 2013
• Last Updated: December 11, 2019

Record last verified: 2019-12

Locations

US (1)