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

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2016 by Lahey Clinic
Information provided by (Responsible Party):
Lahey Clinic Identifier:
First received: March 30, 2009
Last updated: June 22, 2016
Last verified: June 2016
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 Intervention
Lambert-Eaton Myasthenic Syndrome
Congenital Myasthenic Syndrome
Drug: 3,4-Diaminopyridine

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: 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)

Resource links provided by NLM:

Further study details as provided by Lahey Clinic:

Primary Outcome Measures:
  • Increase in strength and autonomic symptoms [ Time Frame: 1-10 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 25
Study Start Date: June 2001
Estimated Study Completion Date: June 2020
Estimated Primary Completion Date: June 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00872950

Contact: Nick F Ventura 781-744-3216
Contact: Stephanie A Scala, MA 781-744-2950

United States, Massachusetts
Lahey Clinic Recruiting
Burlington, Massachusetts, United States, 01805
Contact: Lynn A Sullivan    781-744-2958   
Contact: Stephanie A Scala, MA    781-744-2950   
Sponsors and Collaborators
Lahey Clinic
Principal Investigator: Jayashri Srinivasan, MD, MCRP, PhD Lahey Clinic
Study Director: H. Royden Jones, MD Lahey Clinic
  More Information

Additional Information:
Responsible Party: Lahey Clinic Identifier: NCT00872950     History of Changes
Other Study ID Numbers: 2001-040 
Study First Received: March 30, 2009
Last Updated: June 22, 2016
Health Authority: United States: Food and Drug Administration

Keywords provided by Lahey Clinic:
3,4 DAP

Additional relevant MeSH terms:
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
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action processed this record on October 21, 2016