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Controlled Trial of 3,4-Diaminopyridine (3-4DAP) in Lambert-Eaton Myasthenic Syndrome (LEMS) (3-4DAP)

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ClinicalTrials.gov Identifier: NCT02090725
Recruitment Status : Enrolling by invitation
First Posted : March 18, 2014
Last Update Posted : August 2, 2016
Jacobus Pharmaceutical
Information provided by (Responsible Party):
Dartmouth-Hitchcock Medical Center

Brief Summary:
The main purpose for this study is to provide access to 3,4 DAP, a drug which has demonstrated to be effective in treating weakness associated with Lambert-Eaton Myasthenic Syndrome. LEMS is a rare autoimmune cause of a defect in neuromuscular transmission. The disorder is clinically characterized by fluctuating muscle weakness, hyporeflexia and autonomic dysfunction.

Condition or disease Intervention/treatment
Muscle Weakness Drug: 3-4 Diaminopyridine

Detailed Description:

More than half of LEMS cases are associated with malignancy, usually small cell lung cancer. These paraneoplastic cases progress more quickly than primary autoimmune LEMS. An overlap syndrome with other autoimmune diseases is often detected in LEMS patients.

3,4 DAP is effective in LEMS because it increases calcium influx into the nerve terminal by blocking potassium efflux and thereby prolonging the presynaptic action potential. 3,4 DAP is less likely to provoke epileptic seizures than its precursor, 4-aminopyridine, because it is less able to cross the blood-brain barrier. 3,4 DAP is effective in increasing strength and improving autonomic symptoms in LEMS patients of both the primary autoimmune and paraneoplastic etiologies.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Controlled Trial of 3,4-Diaminopyridine in LEMS
Study Start Date : February 2004
Estimated Primary Completion Date : December 2020

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: 3-4 Diaminopyridine (DAP) Drug: 3-4 Diaminopyridine
Other Name: 3-4 DAP

Primary Outcome Measures :
  1. Change in muscle weakness [ Time Frame: Month 1, Month 2, Month 3, then changing to every 6 months once patient is stable ]
    Muscle weakness will be assessed monthly for the first 3 months based on office visits and blood tests (complete blood count (CBC), electrolytes, glucose, liver function tests, blood urea nitrogen (BUN), creatinine). Muscle weakness will then be assessed every 6 months once the patient is stabilized based on office visits and blood tests (CBC, electrolytes, glucose, liver function tests, BUN, creatinine).

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Ages Eligible for Study:   45 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria: -Male or female majority between 45 and 60 years of age

  • diagnosed with Lambert-Eaton Myasthenic Syndrome.
  • subjects must be taking full dose of pyridostigmine

Exclusion Criteria: - does subject have a history of liver problems?

  • does subject have a history of prolonged QTc syndrome (which is a condition where there is prolongation between the start of the Q wave and the end of the T wave in the heart's electrical cycle).

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): NCT02090725

United States, New Hampshire
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, United States, 03221
Sponsors and Collaborators
Dartmouth-Hitchcock Medical Center
Jacobus Pharmaceutical
Principal Investigator: Jeffrey A. Cohen, MD Dartmouth-Hitchcock Medical Center

Responsible Party: Dartmouth-Hitchcock Medical Center
ClinicalTrials.gov Identifier: NCT02090725     History of Changes
Other Study ID Numbers: 16767
First Posted: March 18, 2014    Key Record Dates
Last Update Posted: August 2, 2016
Last Verified: August 2016

Keywords provided by Dartmouth-Hitchcock Medical Center:
fluctuating muscle weakness
autonomic Dysfunction

Additional relevant MeSH terms:
Lambert-Eaton Myasthenic Syndrome
Paraneoplastic Syndromes, Nervous System
Paraneoplastic Syndromes
Muscle Weakness
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Signs and Symptoms
Nervous System Neoplasms
Neoplasms by Site
Autoimmune Diseases of the Nervous System
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action