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

• Conditions: Myasthenia gravis; MedDRA version: 7.1Level: LLTClassification code 10028417

• Registration Number: EUCTR2004-000596-34-CZ
• Lead Sponsor: F.Hoffmann-La Roche Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2005-05-06
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 136

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: is to assess the efficacy of mycophenolate mofetil therapy compared to placebo in myasthenia gravis patients receiving prednisone;Secondary Objective: is to assess the safety and tolerability of mycophenolate mofetil therapy compared to placebo in mysthenia 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:<br>Minimal Manifestations or Pharmacologic Remission (MGFA Postintervention Status definitions modified) from Week 32 until study termination at Week 36 AND<br>Prednisone dose of not more than 7.5 mg/day from Week 32 until<br>study termination at Week 36 AND<br>Cholinesterase inhibitor dose of =120 mg/day from Week 33* until study termination at Week 36<br>*subjects have one week to reduce cholinesterase inhibitor dose<br>after reaching 7.5mg/day prednisone<br>