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Mycophenolate Mofetil in Myasthenia Gravis

Primary Purpose

Myasthenia Gravis

Status
Completed
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
mycophenolate mofetil
Sponsored by
FDA Office of Orphan Products Development
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myasthenia Gravis focused on measuring myasthenia gravis

Eligibility Criteria

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

Inclusion criteria Acquired generalized MG diagnosed by one of the Principal Investigators based on: Examination by site PI showing myasthenic weakness that is not limited to the ocular or peri-ocular muscles. Elevated acetylcholine receptor antibodies. Positive edrophonium chloride test or abnormal neuromuscular transmission demonstrated by single fiber EMG or repetitive nerve stimulation. Aged at least 18. Able to give informed consent. Taking a constant dose of Mestinon for at least 2 weeks. Symptom severity that would, in the judgment of the site investigator, justify initiation of immunosuppressive treatment. Able and willing to comply with study requirements. Exclusion criteria Thymoma now or in the past. Plasma exchange or IVIG treatment within 90 days of randomization. Treatment with azathioprine, cyclosporine, mycophenolate mofetil, or other immunosuppressive medication since onset of MG. Treatment with prednisone or other corticosteroids within the previous 90 days. • Exception: patients may have taken doses of these immunosuppressant medications that are judged by the Principal Investigator to have been clinically insignificant, i.e. unlikely to produce improvement in MG. Women of childbearing potential who are pregnant, breast-feeding or not practicing effective contraception. Renal failure, active thyroid or hepatocellular disease, chronic infection, poorly controlled cardiac disease, or any other illness, including psychiatric disease, that would, in the opinion of the treating physician, make it unsafe for the patient to participate or would interfere with the interpretation of study results. Weakness affecting only ocular or peri-ocular muscles (Myasthenia Gravis Foundation of America Class I). Severe weakness predominantly affecting oropharyngeal, respiratory muscles or both (MGFA Class IVB). Crisis or impending crisis (defined as FVC <10ml/Kg or bulbar weakness severe enough to compromise airway protection.) Hemoglobin <10mg/dl; WBC <3,500. History of non-compliance with treatment and office visits. Thymectomy within 12 months before randomization. Concurrent medical condition that would pose an unacceptable risk from immunosuppression, including a positive skin test for tuberculosis (PPD), unless the patient has previously received appropriate treatment.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    QMGS

    Secondary Outcome Measures

    Multiple

    Full Information

    First Posted
    January 31, 2006
    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
    NCT00285350
    Brief Title
    Mycophenolate Mofetil in Myasthenia Gravis
    Official Title
    A Trial of Mycophenolate Mofetil in Myasthenia Gravis
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    March 2007
    Overall Recruitment Status
    Completed
    Study Start Date
    September 2002 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    March 2007 (Actual)

    3. Sponsor/Collaborators

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

    4. Oversight

    5. Study Description

    Brief Summary
    This is a prospective, multi-center, double-blind, placebo-controlled trial to determine the efficacy and safety of mycophenolate mofetil (MM) in combination with prednisone as the initial form of immunosuppression in patients with acquired myasthenia gravis (MG).
    Detailed Description
    80 patients with seropositive MG at 18 academic centers will be randomized to 3 months of treatment with 2.5 gm MM/day (1,250 mg q 12 hours, +/- 2 hours) plus 20 mg prednisone/day versus placebo plus 20 mg/day prednisone. The primary measure of efficacy will be the change from baseline in Quantitative MG (QMG) score at the end of 3 months. Secondary outcome measures include survival analysis for treatment failure, MG-related impairment of daily activities, functional assessment, manual muscle testing, SF-36 Health Status, and serum concentration of antibodies to the acetylcholine receptor. Study completers will have the option of taking open-label MM for an additional 6 months, during which prednisone will be reduced to the lowest dose necessary to maintain the optimum clinical response.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Myasthenia Gravis
    Keywords
    myasthenia gravis

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Parallel Assignment
    Masking
    Double
    Allocation
    Randomized
    Enrollment
    80 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    mycophenolate mofetil
    Primary Outcome Measure Information:
    Title
    QMGS
    Secondary Outcome Measure Information:
    Title
    Multiple

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion criteria Acquired generalized MG diagnosed by one of the Principal Investigators based on: Examination by site PI showing myasthenic weakness that is not limited to the ocular or peri-ocular muscles. Elevated acetylcholine receptor antibodies. Positive edrophonium chloride test or abnormal neuromuscular transmission demonstrated by single fiber EMG or repetitive nerve stimulation. Aged at least 18. Able to give informed consent. Taking a constant dose of Mestinon for at least 2 weeks. Symptom severity that would, in the judgment of the site investigator, justify initiation of immunosuppressive treatment. Able and willing to comply with study requirements. Exclusion criteria Thymoma now or in the past. Plasma exchange or IVIG treatment within 90 days of randomization. Treatment with azathioprine, cyclosporine, mycophenolate mofetil, or other immunosuppressive medication since onset of MG. Treatment with prednisone or other corticosteroids within the previous 90 days. • Exception: patients may have taken doses of these immunosuppressant medications that are judged by the Principal Investigator to have been clinically insignificant, i.e. unlikely to produce improvement in MG. Women of childbearing potential who are pregnant, breast-feeding or not practicing effective contraception. Renal failure, active thyroid or hepatocellular disease, chronic infection, poorly controlled cardiac disease, or any other illness, including psychiatric disease, that would, in the opinion of the treating physician, make it unsafe for the patient to participate or would interfere with the interpretation of study results. Weakness affecting only ocular or peri-ocular muscles (Myasthenia Gravis Foundation of America Class I). Severe weakness predominantly affecting oropharyngeal, respiratory muscles or both (MGFA Class IVB). Crisis or impending crisis (defined as FVC <10ml/Kg or bulbar weakness severe enough to compromise airway protection.) Hemoglobin <10mg/dl; WBC <3,500. History of non-compliance with treatment and office visits. Thymectomy within 12 months before randomization. Concurrent medical condition that would pose an unacceptable risk from immunosuppression, including a positive skin test for tuberculosis (PPD), unless the patient has previously received appropriate treatment.
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Donald B Sanders, MD
    Organizational Affiliation
    Duke University
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

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