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

This study has been completed.

Sponsors and Collaborators: FDA Office of Orphan Products Development
Duke University
Information provided by: FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier: NCT00004832
  Purpose

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 Intervention
Lambert-Eaton Myasthenic Syndrome
Drug: 3,4-diaminopyridine

ChemIDplus related topics:   3,4-Diaminopyridine   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment, Randomized, Double-Blind, Placebo Control

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment:   26
Study Start Date:   August 1994
Estimated Study Completion Date:   June 1998

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.

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

Criteria

PROTOCOL ENTRY CRITERIA:

--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

  Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004832

Sponsors and Collaborators

Investigators
Study Chair:     Donald B. Sanders     Duke University    
  More Information


Study ID Numbers:   199/13441, DUMC-577, DUMC-FDR001068
First Received:   February 24, 2000
Last Updated:   June 23, 2005
ClinicalTrials.gov Identifier:   NCT00004832
Health Authority:   United States: Federal Government

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

Study placed in the following topic categories:
Autoimmune Diseases
Paraneoplastic Syndromes
Neuromuscular Diseases
Mental Disorders
3,4-diaminopyridine
Rare Diseases
4-Aminopyridine
Lambert-Eaton Myasthenic Syndrome
Neurodegenerative Diseases
Autoimmune Diseases of the Nervous System
Nervous System Neoplasms

Additional relevant MeSH terms:
Disease
Immune System Diseases
Molecular Mechanisms of Pharmacological Action
Nervous System Diseases
Cardiovascular Agents
Pharmacologic Actions
Membrane Transport Modulators
Neoplasms
Neoplasms by Site
Pathologic Processes
Therapeutic Uses
Syndrome
Potassium Channel Blockers
Neuromuscular Junction Diseases
Paraneoplastic Syndromes, Nervous System

ClinicalTrials.gov processed this record on October 07, 2008




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