Mycophenolate Mofetil in Myasthenia Gravis

Phase 3

Completed

• Conditions: Myasthenia Gravis

• Registration Number: NCT00285350
• Lead Sponsor: FDA Office of Orphan Products Development

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.

Study Timeline

• Study Start: 2002-09
• Study First Submitted: 2006-01-31
• Study First Submitted QC: 2006-01-31
• Study First Posted: 2006-02-02
• Status Verified: 2007-03
• Study Completion: 2007-03
• Last Update Submitted: 2015-03-24
• Last Update Posted: 2015-03-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 80

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
QMGS

Secondary Outcome Measures

Name	Time	Method
Multiple