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

Primary Purpose

Lambert-Eaton Myasthenic Syndrome

Status
Completed
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
3,4-diaminopyridine
Sponsored by
FDA Office of Orphan Products Development
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Lambert-Eaton Myasthenic Syndrome focused on measuring Lambert-Eaton myasthenic syndrome, neurologic and psychiatric disorders, rare disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    February 24, 2000
    Last Updated
    March 24, 2015
    Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    Duke University
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00004832
    Brief Title
    Randomized Study of 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    July 1998
    Overall Recruitment Status
    Completed
    Study Start Date
    August 1994 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    June 1998 (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    Duke University

    4. Oversight

    5. Study Description

    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.
    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Lambert-Eaton Myasthenic Syndrome
    Keywords
    Lambert-Eaton myasthenic syndrome, neurologic and psychiatric disorders, rare disease

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Not Applicable
    Masking
    Double
    Allocation
    Randomized
    Enrollment
    26 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    3,4-diaminopyridine

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility 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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Donald B. Sanders
    Organizational Affiliation
    Duke University
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Learn more about this trial

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

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