Rituximab in Dermatomyositis
Study Title:
An open label trial to evaluate the safety and efficacy of rituximab in the treatment of patients with dermatomyositis
Principal Investigator:
David Fiorentino, MD, PhD
Study Hypothesis:
B cell depletion with Rituximab will be safe and effective therapy for skin and muscle inflammation in patients with dermatomyositis
Study Design:
Open label, single center, pilot study
Types & Number of IIM Studied:
8 adult dermatomyositis patients
Entry Criteria:
- Diagnosis of probable or definite dermatomyositis by Bohan/Peter criteria (Bohan and Peter 1975), including classic dermatologic features defined by Sontheimer's criteria
- Active muscle disease with either elevated laboratory markers of muscle breakdown (ie. CPK >400 U/L or aldolase >8 U/L) OR a MMT modified MRC score of <85
- Active skin disease with s DSSI>2
IMACS Core Set Measures Included:
Physician Global Activity-VAS, Patient Global Activity-VAS, Muscle Strength Testing-MMT, Functional Assessment Tools-HAQ, Laboratory-Muscle Enzymes, Extramuscular Assessment-Myositis Disease Activity Assessment Tool (MDAAT)
Other Measures Included:
Patient Pruritus Assessment, Dermatomyositis Skin Severity Index (DSSI), Skin photographs, CD19 B cell level, Safety laboratory measures (CBC, CMP, Urinalysis)
Primary Outcome:
- Safety Endpoints
- Incidence of treatment emergent adverse events (AE's) including infections, infusion reactions and disease progression.
- Safety assessment will include physical examination with vital signs, routine blood work (hematology and chemistry) and immunological testing.
- Primary Efficacy Endpoint: Partial remission (PR) at 24 weeks, defined as:
- At least a 50% reduction in creatine phosphokinase (CPK) if baseline values are >2x upper limits of normal OR
- 50% improvement of manual muscle strength test (MMT) using the modified Medical Research Council (MRC) scale (Daniels and Worthingham 1972), defined as a 50% reduction in the difference between maximum strength (score of 90) and the patient's baseline score (if baseline score is <85) OR
- 75% reduction in cutaneous lesions using the Dermatomyositis Skin Severity Index (DSSI) if baseline score is > 2
Key Trial Dates:
Start of Enrollment - 12/17/2004
Study Completion - 8/28/2007
Funding Sources:
Genentech, Inc.
Contact:
-
David Fiorentino, M.D., Ph.D.
Assistant Professor of Dermatology -
Stanford University School of Medicine
North Campus Outpatient Center
450 Broadway, C-234
Redwood City, CA 94063
Tel (650) 721-7160
fiorentino@stanford.edu
Return to Therapeutic Studies listing
