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Trial record 4 of 12 for:    Congenital Myasthenia

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT00872950
Recruitment Status : Recruiting
First Posted : April 1, 2009
Last Update Posted : June 15, 2018
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 Drug: 3,4-Diaminopyridine Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
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 2020
Estimated Study Completion Date : June 2020

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

Drug: 3,4-Diaminopyridine
Other Name: 3,4-DAP

Primary Outcome Measures :
  1. Increase in strength and autonomic symptoms [ Time Frame: 1-10 years ]

Information from the National Library of Medicine

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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

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Contact: Erin E Clark 781-744-2862
Contact: LeeAnne Lipert 781-744-8383

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United States, Massachusetts
Lahey Hospital & Medical Center Recruiting
Burlington, Massachusetts, United States, 01805
Contact: Erin E Clark    781-744-2862   
Sponsors and Collaborators
Lahey Clinic
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Principal Investigator: Jayashri Srinivasan, MD, MCRP, PhD Lahey Clinic

Additional Information:
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Responsible Party: Lahey Clinic Identifier: NCT00872950     History of Changes
Other Study ID Numbers: 2001-040
First Posted: April 1, 2009    Key Record Dates
Last Update Posted: June 15, 2018
Last Verified: June 2018

Keywords provided by Lahey Clinic:
3,4 DAP

Additional relevant MeSH terms:
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Myasthenic Syndromes, Congenital
Lambert-Eaton Myasthenic Syndrome
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