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.

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2004-000596-34-HU
• Date of registration: 08/06/2005
• Prospective Registration: Yes
• Primary sponsor: F. Hoffmann-La Roche Ltd as part of the Aspreva Rare Disease Program
• Public title: 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.
• Scientific title: 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.
• Date of first enrolment: 30/09/2005
• Target sample size: 136
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2004-000596-34
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: Single blind: Double blind: yes Parallel group: yes Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: yes Other:

Countries of recruitment

Czech Republic, Hungary, Italy, Spain

Key inclusion & exclusion criteria

Inclusion criteria:
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

Exclusion criteria:
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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Myasthenia gravis
MedDRA version: 7 Level: PT Classification code 10028417

Intervention(s)

Trade Name: CellCept®500 mg tablets
Product Name: CellCept 500 mg film-coated tablets
Pharmaceutical Form: Tablet
INN or Proposed INN: mycophenolate mofetil
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 500-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Secondary Objective: to assess the safety and tolerability of mycophenolate mofetil therapy compared to placebo in myasthenia gravis patients receiving prednisone.

Main Objective: to assess the efficacy 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:

- Minimal Manifestations or Pharmacologic Remission (MGFA Postintervention Status definitions modified) from Week 32 until study termination at Week 36 and,

- Prednisone dose of not more than 7.5 mg/day from Week 32 until study termination at Week 36 and,

-Cholinesterase inhibitor dose of =120 mg/day from Week 33* until study termination at Week 36

*patients have one week to reduce cholinesterase inhibitor dose after reaching 7.5 mg/day prednisone

Secondary ID(s)

2004-000596-34-CZ

WX17798