A prospective, randomized, double-blind, placebo controlled, parallel group, multicenter, 36-weeks trial to assess the efficacy and safety of adjunct mycophenolate mofetil (MMF) to maintain or improve symptoms control with reduced corticosteroids in subjects with mysthenia gravis

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2004-000596-34-CZ
• Date of registration: 06/05/2005
• Prospective Registration: Yes
• Primary sponsor: F.Hoffmann-La Roche Ltd.
• Public title: A prospective, randomized, double-blind, placebo controlled, parallel group, multicenter, 36-weeks trial to assess the efficacy and safety of adjunct mycophenolate mofetil (MMF) to maintain or improve symptoms control with reduced corticosteroids in subjects with mysthenia gravis
• Scientific title: A prospective, randomized, double-blind, placebo controlled, parallel group, multicenter, 36-weeks trial to assess the efficacy and safety of adjunct mycophenolate mofetil (MMF) to maintain or improve symptoms control with reduced corticosteroids in subjects with mysthenia gravis
• Date of first enrolment: 19/05/2005
• 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: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no

Countries of recruitment

Czech Republic, Hungary, Italy, Spain

Key inclusion & exclusion criteria

Inclusion criteria:
Subject of either sex, 18 to 80 years of age (inclusive)
diagnosis of MG 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 antibodies
disease severity history: Myasthenia Gravis Foundation of
America (MGFA) classification II, III, or IVa
duration of MG symptoms (including ocular symptoms) = 10
years
requirement of immunosuppressive therapy in the judgment of the
investigator
prednisone dose of =20mg/day (or equivalent alternate day dose)
for at least 4 weeks prior to randomization
if subject is taking a cholinesterase inhibitor (i.e. pyridostigmine
bromide), a stable regimen is required for at least 2 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 regularly scheduled plasma exchange (PE) or
intravenous immunoglobulin (IVIG) treatment or receiving PE or
IVIG treatment within 2 weeks prior to randomization
receiving MMF or other immunosuppressant therapy (except
corticosteroids) within 8 weeks prior to randomization
any prior clinically significant use of MMF or other
immunosuppressant therapy (except corticosteroids)
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:
severe active gastrointestinal disease; persistent severe diarrhea;
gastrointestinal hemorrhage
active unhealed peptic ulcer within 3 months prior to
randomization
immune deficiency
malignancy
lymphoproliferative disease or previous total lymphoid irradiation
chronic or frequent drug-resistant bacterial infections or presence
of active infection requiring antimicrobial treatment
frequent and/or serious viral infection
systemic 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: 7.1 Level: LLT Classification code 10028417

Intervention(s)

Trade Name: CellCept 500 mg
Product Name: CellCept
Pharmaceutical Form: Tablet
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: is 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 or 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
*subjects have one week to reduce cholinesterase inhibitor dose
after reaching 7.5mg/day prednisone

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

Secondary ID(s)

WX17798