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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2013 by Lahey Clinic
Sponsor:
Information provided by (Responsible Party):
Lahey Clinic
ClinicalTrials.gov Identifier:
NCT00872950
First received: March 30, 2009
Last updated: May 22, 2013
Last verified: May 2013
  Purpose

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
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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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 ClinicalTrials.gov identifier: NCT00872950

Contacts
Contact: Nick F Ventura 781-744-3216 Nicholas.F.Ventura@lahey.org
Contact: Stephanie A Scala, MA 781-744-2950 stephanie.a.scala@lahey.org

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

Additional Information:
No publications provided

Responsible Party: Lahey Clinic
ClinicalTrials.gov Identifier: NCT00872950     History of Changes
Other Study ID Numbers: 2001-040
Study First Received: March 30, 2009
Last Updated: May 22, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Lahey Clinic:
LEMS
CMS
3,4 DIAMINOPYRIDINE
3,4 DAP

Additional relevant MeSH terms:
Lambert-Eaton Myasthenic Syndrome
Myasthenic Syndromes, Congenital
Syndrome
Autoimmune Diseases
Autoimmune Diseases of the Nervous System
Disease
Genetic Diseases, Inborn
Immune System Diseases
Neoplasms
Neoplasms by Site
Nervous System Diseases
Nervous System Neoplasms
Neurodegenerative Diseases
Neuromuscular Diseases
Neuromuscular Junction Diseases
Paraneoplastic Syndromes
Paraneoplastic Syndromes, Nervous System
Pathologic Processes

ClinicalTrials.gov processed this record on November 23, 2014