Mycophenolate Mofetil (MMF) for Treatment of Chronic Graft-versus-host Disease (GVHD)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00089141|
Recruitment Status : Terminated (Low probability of positive outcome)
First Posted : August 5, 2004
Results First Posted : August 26, 2009
Last Update Posted : May 3, 2013
RATIONALE: Mycophenolate mofetil added to immunosuppressive treatment regimens may be effective in treating newly diagnosed chronic graft-versus-host disease caused by stem cell transplantation. It is not yet known whether immunosuppressive treatment regimens are more effective with or without mycophenolate mofetil in treating chronic graft-versus-host disease.
PURPOSE: This randomized phase III trial is studying whether the addition of mycophenolate mofetil improves the efficacy of immunosuppressive treatment regimens in patients with newly diagnosed chronic graft-versus-host disease.
|Condition or disease||Intervention/treatment||Phase|
|Cancer||Drug: mycophenolate mofetil Drug: placebo||Phase 3|
- 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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||151 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||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|
|Study Start Date :||May 2004|
|Actual Primary Completion Date :||July 2008|
|Actual Study Completion Date :||September 2008|
Active Comparator: Mycophenolate mofetil
Patients receive oral mycophenolate mofetil twice daily.
Drug: mycophenolate mofetil
Other Name: CellCept
Placebo Comparator: Placebo
Patients receive oral placebo twice daily
Other Name: Control
- Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy [ Time Frame: 2 years ]Withdrawal of all systemic immunosuppressive treatment after resolution of chronic GVHD, before death or onset of recurrent malignancy
- Definitive Absence of Efficacy Success [ Time Frame: 2 years ]Administration of secondary systemic therapy for chronic GVHD, death during primary therapy, or onset of recurrent malignancy or bronchiolitis obliterans during primary therapy
- Open Label Systemic Treatment Because of Inadequate Response to Primary Therapy [ Time Frame: 2 years ]Administration of any systemic therapy other than the immunosuppressive agents used for initial treatment, because of persistent or progressive chronic graft-versus-host disease
- Bronchiolitis Obliterans [ Time Frame: within 4 years ]Development of bronchiolitis obliterans during treatment
- Recurrent Malignancy [ Time Frame: within 4 years ]Development of recurrent malignancy after enrollment in the study
- Non-relapse Mortality [ Time Frame: within 4 years ]Death without prior development of recurrent malignancy
- Death or Recurrent Malignancy [ Time Frame: within 4 years ]Death due to any cause or development of recurrent malignancy at any time after enrollment
- Death [ Time Frame: within 4 years ]Death from any cause after enrollment in the study
- Withdrawal of Prednisone [ Time Frame: within 4 years ]Withdrawal of treatment with prednisone after improvement or resolution of chronic GVHD
- End of Systemic Treatment [ Time Frame: within 4 years ]Withdrawal of all immunosuppressive treatment without recurrent malignancy
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00089141
|United States, California|
|City of Hope Comprehensive Cancer Center|
|Duarte, California, United States, 91010-3000|
|Stanford Cancer Center|
|Stanford, California, United States, 94305-5824|
|United States, Florida|
|University of Florida Shands Cancer Center|
|Gainesville, Florida, United States, 32610-100277|
|United States, Illinois|
|University of Chicago Cancer Research Center|
|Chicago, Illinois, United States, 60637-1470|
|United States, Michigan|
|University of Michigan Comprehensive Cancer Center|
|Ann Arbor, Michigan, United States, 48109-0942|
|United States, Minnesota|
|Masonic Cancer Center at University of Minnesota|
|Minneapolis, Minnesota, United States, 55455|
|United States, Nebraska|
|UNMC Eppley Cancer Center at the University of Nebraska Medical Center|
|Omaha, Nebraska, United States, 68198-3330|
|United States, New Jersey|
|Hackensack University Medical Center Cancer Center|
|Hackensack, New Jersey, United States, 07601|
|United States, Oregon|
|Oregon Health and Science University Cancer Institute|
|Portland, Oregon, United States, 97239-3098|
|United States, Tennessee|
|Vanderbilt-Ingram Cancer Center|
|Nashville, Tennessee, United States, 37232-6838|
|United States, Texas|
|Baylor University Medical Center - Dallas|
|Dallas, Texas, United States, 75246|
|M. D. Anderson Cancer Center at University of Texas|
|Houston, Texas, United States, 77030-4009|
|Texas Transplant Institute|
|San Antonio, Texas, United States, 78229|
|United States, Washington|
|Fred Hutchinson Cancer Research Center|
|Seattle, Washington, United States, 98109-1024|
|University of Washington School of Medicine|
|Seattle, Washington, United States, 98195|
|Princess Margaret Hospital|
|Toronto, Ontario, Canada, M5G 2M9|
|Principal Investigator:||Paul J. Martin, MD||Fred Hutchinson Cancer Research Center|