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Mycophenolate Mofetil in Myasthenia Gravis

This study has been completed.
Duke University
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: January 31, 2006
Last updated: March 24, 2015
Last verified: March 2007
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).

Condition Intervention Phase
Myasthenia Gravis Drug: mycophenolate mofetil Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Treatment
Official Title: A Trial of Mycophenolate Mofetil in Myasthenia Gravis

Resource links provided by NLM:

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

Primary Outcome Measures:
  • QMGS

Secondary Outcome Measures:
  • Multiple

Estimated Enrollment: 80
Study Start Date: September 2002
Study Completion Date: March 2007
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.

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

Inclusion criteria

  1. 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.
  2. Aged at least 18.
  3. Able to give informed consent.
  4. Taking a constant dose of Mestinon for at least 2 weeks.
  5. Symptom severity that would, in the judgment of the site investigator, justify initiation of immunosuppressive treatment.
  6. Able and willing to comply with study requirements.

Exclusion criteria

  1. Thymoma now or in the past.
  2. Plasma exchange or IVIG treatment within 90 days of randomization.
  3. 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.

  4. Women of childbearing potential who are pregnant, breast-feeding or not practicing effective contraception.
  5. 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.
  6. Weakness affecting only ocular or peri-ocular muscles (Myasthenia Gravis Foundation of America Class I).
  7. Severe weakness predominantly affecting oropharyngeal, respiratory muscles or both (MGFA Class IVB).
  8. Crisis or impending crisis (defined as FVC <10ml/Kg or bulbar weakness severe enough to compromise airway protection.)
  9. Hemoglobin <10mg/dl; WBC <3,500.
  10. History of non-compliance with treatment and office visits.
  11. Thymectomy within 12 months before randomization.
  12. 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00285350

Sponsors and Collaborators
FDA Office of Orphan Products Development
Duke University
Principal Investigator: Donald B Sanders, MD Duke University
  More Information Identifier: NCT00285350     History of Changes
Obsolete Identifiers: NCT00127894
Other Study ID Numbers: 2154
Study First Received: January 31, 2006
Last Updated: March 24, 2015

Keywords provided by FDA Office of Orphan Products Development:
myasthenia gravis

Additional relevant MeSH terms:
Muscle Weakness
Myasthenia Gravis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Signs and Symptoms
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Mycophenolic Acid
Mycophenolate mofetil
Antibiotics, Antineoplastic
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs processed this record on September 19, 2017