Treatment of Lambert-Eaton Syndrome With 3,4 DAP

NCT00704925 | Unknown

• 1 other identifier: 200311036
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

A new drug called 3,4-Diaminopyridine (3,4-DAP) is currently under investigation for treatment of the symptoms of Lambert-Eaton Myasthenic Syndrome (LEMS). This is an expanded access trial, which means that although data from this study will be collected and reported to the US Food and Drug Administration (FDA)and the drug manufacturer, this is not a formal study of drug in LEMS. If you decide volunteer, you will be evaluated by a neurologist to determine your eligibility to receive 3, 4-DAP by a review of your medical history, medication regimen (the medications you are taking) and a neurological examination. If you are a female of child-bearing potential, a serum pregnancy test will be done to ensure that you are not pregnant. Once it is determined that this treatment is appropriate for your care, you will begin taking 3, 4 DAP by mouth in slowly increasing doses. Treatment will begin with 5mg three times a day, as clinically needed, and if tolerated. You will be monitored for strength and side effects by routine clinic visits at initial intervals of once a month, increasing to intervals of every 12 months as permitted. Blood will be drawn (approximately 1 tablespoon) at every clinic visit or as often as the investigator deems necessary to assess your liver/kidney function and blood counts. You will have an EKG (a test to see how your heart is functioning) at your first study visit, after 6 months of taking 3,4 DAP and again every 2 years. Treatment will be continued indefinitely if a good clinical response is achieved. This study is planned to last indefinitely. The dosage of 3, 4DAP is individually adjusted. The usual range is 10-15 mg 3-4 times per day for the full effect and will increase by 50% every two weeks to 10-15 mg three to six times a day, as needed and if tolerated. Dosages above the full effect level will not provide an additional benefit and should not be used. 3, 4 DAP is a convulsant (causes seizures). A total of 100 mg/day is the maximum dosage allowed.

Conditions

Lambert Eaton Myasthenic Syndrome

Keywords

Lambert Eaton Myasthenic Syndrome; 3,4 DAP; 3,4 Diaminopyridine

Interventions

3, 4 DAP DRUG

Patients with clinically-confirmed LEMS will receive 3, 4 DAP by mouth in slowly increasing doses. Treatment will begin with 5 mg three times a day and will increase by 50% every two weeks to 10-15 mg three to six times a day, as clinically needed, and if tolerated.

Also known as: 3,4 diaminopyridine

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Be 18 years or older, diagnosed with LEMS
• If female, have negative pregnancy test and if premenopausal, be willing to practice an effective form of birth control during the study.

You may not qualify if:

• Is known to have a sensitivity to 3, 4 DAP
• Has a history of past or current seizures
• Has a history of past or current severe asthma
• Is believed by the investigator to be unable to comply with the protocol
• Unable to give informed consent

Sponsors & Collaborators

• David P. Richman, MD: lead
• Jacobus Pharmaceutical: collaborator

Study Sites (1)

University of California, Davis

Sacramento, California, 95817, United States

Location

MeSH Terms

Conditions

Lambert-Eaton Myasthenic Syndrome

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

Intervention Hierarchy (Ancestors)

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

Study Officials

• David Richman, MD

  University of California, Davis

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator, Professor

Study Record Dates

• First Submitted: June 23, 2008
• First Posted: June 25, 2008
• Last Updated: October 30, 2019

Record last verified: 2019-10

Locations

US (1)