A Randomized Study to Evaluate The Efficacy of Mycophenolate Mofetil Added to The Systemic Immunosuppressive Regimen First Used For Treatment of Chronic Graft-Versus-Host Disease
- Compare the efficacy of immunosuppressive treatment regimens with vs without
mycophenolate mofetil in patients with newly diagnosed chronic graft-vs-host disease.
- Compare the quality of life of patients treated with these regimens.
OUTLINE: This is a randomized, double-blind, placebo-controlled, prospective, multicenter
study. Patients are stratified according to organ involvement of chronic graft-versus-host
disease (GVHD) (single organ vs multiple organs) and transplant center. Patients are
randomized to 1 of 2 treatment arms.
All patients receive usual therapy for chronic GVHD comprising oral prednisone twice daily
and oral cyclosporine, oral tacrolimus or oral sirolimus twice daily until 2 weeks after the
first evidence of improvement of symptoms of chronic GVHD.
- Arm I: Patients receive oral mycophenolate mofetil twice daily.
- Arm II: Patients receive oral placebo twice daily. In both arms administration of the
study drug continues for 3 months after completion of prednisone and cyclosporine,
tacrolimus or sirolimus in the absence of disease progression or unacceptable toxicity.
Quality of life is assessed at baseline and then every 3 months.
Patients are followed every 3 months for 3-5 years.
PROJECTED ACCRUAL: A total of 230 patients (115 per treatment arm) will be accrued for this
study within 3 years.
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy
Withdrawal of all systemic immunosuppressive treatment after resolution of chronic GVHD, before death or onset of recurrent malignancy
Paul J. Martin, MD
Fred Hutchinson Cancer Research Center
United States: Food and Drug Administration
|Fred Hutchinson Cancer Research Center||Seattle, Washington 98109|
|University of Michigan Comprehensive Cancer Center||Ann Arbor, Michigan 48109-0752|
|University of Chicago Cancer Research Center||Chicago, Illinois 60637|
|Vanderbilt-Ingram Cancer Center||Nashville, Tennessee 37232-6838|
|City of Hope Comprehensive Cancer Center||Duarte, California 91010|
|University of Florida Shands Cancer Center||Gainesville, Florida 32610-0232|
|Hackensack University Medical Center Cancer Center||Hackensack, New Jersey 07601|
|UNMC Eppley Cancer Center at the University of Nebraska Medical Center||Omaha, Nebraska 68198-7680|
|Masonic Cancer Center at University of Minnesota||Minneapolis, Minnesota 55455|
|M. D. Anderson Cancer Center at University of Texas||Houston, Texas 77030-4009|
|University of Washington School of Medicine||Seattle, Washington 98195|
|Stanford Cancer Center||Stanford, California 94305-5824|
|Texas Transplant Institute||San Antonio, Texas 78229|
|Oregon Health and Science University Cancer Institute||Portland, Oregon 97239-3098|
|Baylor University Medical Center - Dallas||Dallas, Texas 75246|