Randomized Study of 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome

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. Read our disclaimer for details. Identifier: NCT00004832
Recruitment Status : Completed
First Posted : February 25, 2000
Last Update Posted : March 25, 2015
Duke University
Information provided by:
FDA Office of Orphan Products Development

Brief Summary:

OBJECTIVES: I. Evaluate the safety and effectiveness of 3,4-diaminopyridine (DAP) in the treatment of patients with Lambert-Eaton myasthenic syndrome (LEMS).

II. Determine the side-effects and benefits associated with DAP.

Condition or disease Intervention/treatment Phase
Lambert-Eaton Myasthenic Syndrome Drug: 3,4-diaminopyridine Not Applicable

Detailed Description:
PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled study. Patients are randomized to receive 3,4-diaminopyridine (DAP) or placebo orally 3 times daily for 5 days, after which treatment is discontinued and patients are observed for at least 24 hours. At the end of the blinded study, patients may then elect to take open label DAP orally 3 times daily for 6 months; those who do so are monitored for clinical effects and side effects for at least 6 months.

Study Type : Interventional  (Clinical Trial)
Enrollment : 26 participants
Allocation: Randomized
Masking: Double
Primary Purpose: Treatment
Study Start Date : August 1994
Study Completion Date : June 1998

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics-- Lambert-Eaton myasthenic syndrome (LEMS) based on weakness that predominates in proximal limb muscles and electromyography (EMG) findings of small amplitude muscle responses to nerve stimulation, which decrease further during nerve stimulation at 5 Hz and which increase at least 2-fold after maximum voluntary contraction of the muscle for 10-20 seconds Quantified Myasthenia Gravis (QMG) clinical score at least 5 --Prior/Concurrent Therapy-- Chemotherapy: No concurrent chemotherapy Endocrine therapy: Patients receiving immunosuppressants must be on the same dose of medication for at least 3 months prior to study entry Radiotherapy: No concurrent radiotherapy Surgery: No concurrent surgery Other: Patients receiving cholinesterase inhibitors must discontinue the medication at study entry if possible, or else be on the same dose of medication for at least 1 month prior to study entry --Patient Characteristics-- Hematopoietic: No significant hematologic disease Hepatic: No significant hepatic disease Renal: No significant renal disease Cardiovascular: No cardiac arrhythmia or significant cardiac disease Neurologic: No seizure disorder Other: Not pregnant Negative pregnancy test required of fertile women Effective contraception required of fertile women

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): NCT00004832

Sponsors and Collaborators
FDA Office of Orphan Products Development
Duke University
Study Chair: Donald B. Sanders Duke University Identifier: NCT00004832     History of Changes
Other Study ID Numbers: 199/13441
First Posted: February 25, 2000    Key Record Dates
Last Update Posted: March 25, 2015
Last Verified: July 1998

Keywords provided by FDA Office of Orphan Products Development:
Lambert-Eaton myasthenic syndrome
neurologic and psychiatric disorders
rare disease

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