Mycophenolate Mofetil (MMF) for Treatment of Chronic Graft-versus-host Disease (GVHD)

Phase 3

Terminated

Brief Summary: 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.

Detailed Description: OBJECTIVES:

* 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.

Recruitment & Eligibility

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
Mycophenolate mofetil	mycophenolate mofetil	Patients receive oral mycophenolate mofetil twice daily.
Placebo	placebo	Patients receive oral placebo twice daily

Primary Outcome Measures

Name	Time	Method
Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy	2 years	Withdrawal of all systemic immunosuppressive treatment after resolution of chronic GVHD, before death or onset of recurrent malignancy

Secondary Outcome Measures

Name	Time	Method
Definitive Absence of Efficacy Success	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	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	within 4 years	Development of bronchiolitis obliterans during treatment
Recurrent Malignancy	within 4 years	Development of recurrent malignancy after enrollment in the study
Non-relapse Mortality	within 4 years	Death without prior development of recurrent malignancy
Death or Recurrent Malignancy	within 4 years	Death due to any cause or development of recurrent malignancy at any time after enrollment
Death	within 4 years	Death from any cause after enrollment in the study
Withdrawal of Prednisone	within 4 years	Withdrawal of treatment with prednisone after improvement or resolution of chronic GVHD
End of Systemic Treatment	within 4 years	Withdrawal of all immunosuppressive treatment without recurrent malignancy

Locations (16): City of Hope Comprehensive Cancer Center
🇺🇸
Duarte, California, United States
Stanford Cancer Center
🇺🇸
Stanford, California, United States
University of Florida Shands Cancer Center
🇺🇸
Gainesville, Florida, United States
University of Chicago Cancer Research Center
🇺🇸
Chicago, Illinois, United States
University of Michigan Comprehensive Cancer Center
🇺🇸
Ann Arbor, Michigan, United States
Masonic Cancer Center at University of Minnesota
🇺🇸
Minneapolis, Minnesota, United States
UNMC Eppley Cancer Center at the University of Nebraska Medical Center
🇺🇸
Omaha, Nebraska, United States
Hackensack University Medical Center Cancer Center
🇺🇸
Hackensack, New Jersey, United States
Oregon Health and Science University Cancer Institute
🇺🇸
Portland, Oregon, United States
Vanderbilt-Ingram Cancer Center
🇺🇸
Nashville, Tennessee, United States
Scroll for more (6 remaining)
City of Hope Comprehensive Cancer Center
🇺🇸Duarte, California, United States