A prospective, randomized, double-blind, placebo-controlled, parallel group, multicenter, 36-week trial to assess the efficacy and safety of adjunct mycophenolate mofetil (MMF) to maintain or improve symptom control with reduced corticosteoids in subjects with myasthenia gravis.
- Conditions
- MedDRA version: 7Level: PTClassification code 10028417Myasthenia gravis
- Registration Number
- EUCTR2004-000596-34-ES
- Lead Sponsor
- F. Hoffmann-La Roche Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 136
Diagnosis of myasthenia gravis meeting all of the following criteria:
- history of myasthenic weakness involving more than ocular or peri-ocular muscles
- history of positive edrophonium chloride test or abnormal neuromuscular transmission demonstrated by electrodiagnostic testing
- history of elevated AchR antiodies
Disease severity history: MGFA classification II, III or IVa
Duration of MG symptoms (including ocular symptoms) = 10 years
Prednisone dose of = 20 mg/day (or equivalent alternate day dose) for at least 4 weeks prior to randomization
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
Pregnancy, breastfeeding or lactation
Receiving plasma exchange or intravenous immunoglobulin treatment regularly or within 2 weeks prior to randomization
Receiving MMF or other immunosuppressant therapy (except corticosteroids) within 8 weeks prior to randomization
Severe weakness of oropharyngeal and/or respiratory muscles (MGFA Class IVb or V; compromised airway protection; MG crisis or impending crisis)
Thymoma
Thymectomy within 6 months prior to randomization
Presence or history of immune deficiency, malignancy, lymphoproliferative disease or previous total lymphoid irradiation, frequent and/or serious viral infection, systematic or invasive fungal disease within 2 years prior to randomization, significant kidney or liver dysfunction, pulmonary insufficiency requiring supplemental oxygen, bone marrow insufficiency
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: to assess the efficacy of mycophenolate mofetil therapy compared to placebo in myasthenia gravis patients receiving prednisone. ;Secondary Objective: to assess the safety and tolerability of mycophenolate mofetil therapy compared to placebo in myasthenia gravis patients receiving prednisone.;Primary end point(s): Treatment groups will be compared to measure the proportion of subjects reaching responder status. A subject will be considered a responder if he/she meets all the following criteria:<br><br>- Minimal Manifestations or Pharmacologic Remission (MGFA Postintervention Status definitions modified) from Week 32 until study termination at Week 36 and,<br><br>- Prednisone dose of not more than 7.5 mg/day from Week 32 until study termination at Week 36 and,<br><br>-Cholinesterase inhibitor dose of =120 mg/day from Week 33* until study termination at Week 36<br><br>*patients have one week to reduce cholinesterase inhibitor dose after reaching 7.5 mg/day prednisone
- Secondary Outcome Measures
Name Time Method