Working… Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00872950
Recruitment Status : Active, not recruiting
First Posted : April 1, 2009
Last Update Posted : August 11, 2020
Information provided by (Responsible Party):
Lahey Clinic

Brief 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).

Condition or disease Intervention/treatment Phase
Lambert-Eaton Myasthenic Syndrome Congenital Myasthenic Syndrome Drug: 3,4-DIAMINOPYRIDINE Not Applicable

Detailed 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 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-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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 22 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open Label Trial Of 3,4-Diaminopyridine In Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS)
Study Start Date : June 2001
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : June 2021

Arm Intervention/treatment
Open label
Open label
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.
Other Name: 3,4 DAP

Primary Outcome Measures :
  1. Increase in strength and autonomic symptoms [ Time Frame: 1-10 years ]
    Patients will be monitored for strength and side effects. Treatment will be continued indefinitely if a good clinical response is achieved and side effects tolerable.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00872950

Layout table for location information
United States, Massachusetts
Lahey Hospital & Medical Center
Burlington, Massachusetts, United States, 01805
Sponsors and Collaborators
Lahey Clinic
Layout table for investigator information
Principal Investigator: Jayashri Srinivasan, MD,MCRP,PhD Lahey Clinic
Additional Information:
Layout table for additonal information
Responsible Party: Lahey Clinic Identifier: NCT00872950    
Other Study ID Numbers: 2001-040
First Posted: April 1, 2009    Key Record Dates
Last Update Posted: August 11, 2020
Last Verified: August 2020
Keywords provided by Lahey Clinic:
3,4 DAP
Additional relevant MeSH terms:
Layout table for MeSH terms
Lambert-Eaton Myasthenic Syndrome
Myasthenic Syndromes, Congenital
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
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
Neuromuscular Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action