Mycophenolate Mofetil in Myasthenia Gravis

• Register: ClinicalTrials.gov
• Last refreshed on: 7 April 2015
• Main ID: NCT00285350
• Date of registration: 31/01/2006
• Prospective Registration: No
• Primary sponsor: FDA Office of Orphan Products Development
• Public title: Mycophenolate Mofetil in Myasthenia Gravis
• Scientific title: A Trial of Mycophenolate Mofetil in Myasthenia Gravis
• Date of first enrolment: September 2002
• Target sample size: 80
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00285350
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment
• Phase: Phase 3

Contacts

• Name: Donald B Sanders, MD
• Affiliation: Duke University

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Myasthenia Gravis

Intervention(s)

Drug: mycophenolate mofetil

Primary Outcome(s)

QMGS

Secondary Outcome(s)

Multiple

Secondary ID(s)

2154

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Duke University