Clinical trial NCT00683969: A Study to Assess the Effect of CellCept (Mycophenolate Mofetil) and Reduced Corticosteroids in Controlling Symptoms of Myasthenia Gravis
Official 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 Corticosteroid in Subjects With Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Myasthenia Gravis, Generalized
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
Collaborators: name: Aspreva Pharmaceuticals
First posted: 2008-05-26
Enrollment: 136
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The efficacy and safety of CellCept (1g po, bid for 36 weeks) will be assessed in patients with myasthenia gravis receiving prednisone, or other corticosteroids. During the study, patients will undergo gradual corticosteroid dose reduction, if they respond to treatment. The anticipated time on study treatment is 3-12 months, and the target sample size is 100-500 individuals.


Eligibility criteria: Inclusion Criteria:

* adult patients 18 to 80 years of age;
* diagnosis of myasthenia gravis;
* history of myasthenia weakness involving more than ocular (ie eye) or peri-ocular muscle;
* duration of myasthenia gravis symptoms (including ocular symptoms) \<=10 years;
* prednisone dose \>=20 mg/day (or equivalent alternate-day dose) for \>=4 weeks.

Exclusion Criteria:

* female patients who are pregnant, breastfeeding, or lactating;
* regularly scheduled plasma exchange (PE) or intravenous immunoglobulin (IVIG) treatment, or PE or IVIG treatment within 2 weeks prior to randomization;
* any prior clinically significant use of CellCept or other immunosuppressive therapy (except corticosteroids), or within 8 weeks prior to randomization.

Primary outcomes:
measure: Proportion of subjects reaching responder status
timeFrame: Week 36
Secondary outcomes:
measure: Time to start of response
timeFrame: Event driven
measure: Mean and median prednisone dose and cholinesterase inhibitor dose
timeFrame: Week 36
measure: Adverse events, lab parameters, vital signs
timeFrame: Throughout study
Other outcomes:
Locations:
city: Sun City
country: United States
state: Arizona
city: Sacramento
country: United States
state: California
city: Miami
country: United States
state: Florida
city: Kansas City
country: United States
state: Kansas
city: Rochester
country: United States
state: New York
city: Chapel Hill
country: United States
state: North Carolina
city: Columbus
country: United States
state: Ohio
city: Upland
country: United States
state: Pennsylvania
city: Dallas
country: United States
state: Texas
city: Galveston
country: United States
state: Texas
city: Edmonton
country: Canada
state: Alberta
city: London
country: Canada
state: Ontario
city: Belgrade
country: Former Serbia and Montenegro
city: Bordeaux Cedex
country: France
city: Nice
country: France
city: Düsseldorf
country: Germany
city: München
country: Germany
city: Regensburg
country: Germany
city: Tübingen
country: Germany
city: Hyderabad
country: India
city: Mumbai
country: India
city: New Delhi
country: India
city: Jerusalem
country: Israel
city: Milano
country: Italy
city: Roma
country: Italy
city: San Donato Milanese
country: Italy
city: Mexico City
country: Mexico
city: Mexico City
country: Mexico
city: Mexico City
country: Mexico
city: Leiden
country: Netherlands
city: Maastricht
country: Netherlands
city: Rotterdam
country: Netherlands
city: Kazan
country: Russian Federation
city: Moscow
country: Russian Federation
city: Nizhniy Novgorad
country: Russian Federation
city: Barcelona
country: Spain
city: Madrid
country: Spain
city: Kharkov
country: Ukraine
city: Kiev
country: Ukraine
city: Zaporozhye
country: Ukraine
city: Liverpool
country: United Kingdom
city: Oxford
country: United Kingdom
city: Salford
country: United Kingdom
Study officials:
affiliation: Hoffmann-La Roche, 973-235-5000
name: Clinical Trials
role: STUDY_DIRECTOR
References:
citation: Sengupta M, Cheema A, Kaminski HJ, Kusner LL; Muscle Study Group. Serum metabolomic response of myasthenia gravis patients to chronic prednisone treatment. PLoS One. 2014 Jul 17;9(7):e102635. doi: 10.1371/journal.pone.0102635. eCollection 2014.