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

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ClinicalTrials.gov Identifier: NCT00872950
Recruitment Status : Active, not recruiting
First Posted : April 1, 2009
Last Update Posted : August 11, 2020
Sponsor:
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.

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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
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




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.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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

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 ClinicalTrials.gov identifier (NCT number): NCT00872950


Locations
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United States, Massachusetts
Lahey Hospital & Medical Center
Burlington, Massachusetts, United States, 01805
Sponsors and Collaborators
Lahey Clinic
Investigators
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Principal Investigator: Jayashri Srinivasan, MD,MCRP,PhD Lahey Clinic
Additional Information:
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Responsible Party: Lahey Clinic
ClinicalTrials.gov 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:
LEMS
CMS
3,4 DIAMINOPYRIDINE
3,4 DAP
Additional relevant MeSH terms:
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Lambert-Eaton Myasthenic Syndrome
Myasthenic Syndromes, Congenital
Syndrome
Disease
Pathologic Processes
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
Genetic Diseases, Inborn
Amifampridine
Neuromuscular Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action