Mycophenolate Mofetil in Myasthenia Gravis

NCT00285350 | Completed Phase 3

• 1 other identifier: 2154
• Study Type: interventional
• Target: 80
• Locations: 0 countries
• Sites: N/A

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).

Conditions

Myasthenia Gravis

Keywords

myasthenia gravis

Outcome Measures

Primary Outcomes (1)

• QMGS

Secondary Outcomes (1)

• Multiple

Interventions

mycophenolate mofetil DRUG

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• FDA Office of Orphan Products Development: lead
• Duke University: collaborator

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

Mycophenolic Acid

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids

Study Officials

• Donald B Sanders, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: FED

Study Record Dates

• First Submitted: January 31, 2006
• First Posted: February 2, 2006
• Study Start: September 1, 2002
• Study Completion: March 1, 2007
• Last Updated: March 25, 2015

Record last verified: 2007-03