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A Multi-Center, Randomized, Double Blind, Phase III Trial Evaluating Corticosteroids With Mycophenolate Mofetil vs. Corticosteroids With Placebo as Initial Systemic Treatment of Acute GVHD (BMT CTN 0802)

Phase 3
Not Enrolling
Graft-versus-Host Disease, Immune System Disorders

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Trial Information

A Multi-Center, Randomized, Double Blind, Phase III Trial Evaluating Corticosteroids With Mycophenolate Mofetil vs. Corticosteroids With Placebo as Initial Systemic Treatment of Acute GVHD (BMT CTN 0802)

Corticosteroids have been used as primary therapy for acute GVHD for many years. Historical
published and unpublished data from Johns Hopkins, M. D. Anderson, University of Michigan
and others defined an expected 35%-53% complete response (CR) at Day +28 of corticosteroid
therapy for previously untreated patients with acute GVHD.

BMT CTN study 0302 (NCT00224874)was a randomized Phase II study evaluating etanercept,
mycophenolate mofetil, denileukin diftitox or pentostatin in addition to corticosteroids.
The results of that study suggested that mycophenolate mofetil produced the highest rates of
CR at Day 28 and overall survival, supporting its evaluation in a Phase III study. Day 56
GVHD-free survival for the four treatment arms (all combining corticosteroids with one of
the four study drugs) ranged from 39-71% across the four study arms.

Inclusion Criteria:

- Acute GVHD developing after allogeneic hematopoietic stem cell transplant using
either bone marrow, peripheral blood stem cells or cord blood. Recipients of
non-myeloablative and myeloablative transplants are eligible.

- Acute GVHD after planned donor lymphocyte infusion or planned T cell addback are

- De novo acute GVHD requiring systemic therapy. GVHD is defined as the presence of
skin rash and/or persistent nausea, vomiting, and/or diarrhea and/or cholestasis
presenting in a context in which acute GVHD is likely to occur and where other
etiologies such as drug rash, enteric infection, or hepatotoxic syndromes are
unlikely or have been ruled out. Note that patients with stage I and II skin only
(overall grade I) or isolated upper gastrointestinal (GI) involvement are eligible if
the treating physician deems that systemic high-dose corticosteroid treatment is

- The patient must have had no previous systemic immune suppressive therapy for
treatment of acute GVHD except for a maximum 72 hours of prior corticosteroid therapy
at >0.5mg/kg methylprednisolone or equivalent after the onset of acute GVHD.

- Clinical status at enrollment to allow tapering of steroids to not less than 0.25
mg/kg/day prednisone (0.2 mg/kg/day methylprednisolone) at Day 28 of therapy.

- Absolute neutrophil count (ANC) greater than 500/µL.

- Written informed consent and/or assent from patient, parent or guardian.

- Documentation that the assent document and education materials have been provided to,
and reviewed with, patients between the ages of 7 and 17.

- Patients of all ages are eligible.

- Biopsy confirmation of GVHD is recommended, but not required. Enrollment should not
be delayed for biopsy or pathology results unless these are to be used to decide
about whether to treat for GVHD.

Exclusion Criteria:

- Patients receiving mycophenolate mofetil or mycophenolic acid (Myfortic) within seven
days of screening for enrollment.

- Patients with uncontrolled infections will be excluded. If a bacterial or viral
infection is present, patients must be receiving definitive therapy and have no signs
of progressing infection for 72 hours prior to enrollment. If a fungal infection is
present, patients must be receiving definitive systemic anti-fungal therapy and have
no signs of progressing infection for 1 week prior to enrollment. Progressing
infection is defined as hemodynamic instability attributable to sepsis or new
symptoms, worsening physical signs or radiographic findings attributable to
infection. Persisting fever without other signs or symptoms will not be interpreted
as progressing infection.

- Relapsed/persistent malignancy requiring rapid immune suppression withdrawal.

- Patients with GVHD after an unplanned DLI, i.e., DLI that was not part of their
original transplant therapy plan, or DLI given for treatment of persistent or
recurrent malignancy after transplantation.

- Patients unlikely to be available at the transplantation center on Day 28 and 56 of

- A clinical syndrome resembling de novo chronic GVHD developing at any time after

- Patients receiving other drugs for the treatment of GVHD.

- Patients receiving methylprednisolone > 0.5 mg/kg/day (or 0.6 mg/kg/day prednisone)
within 7 days before the onset of acute GVHD. If steroid therapy has been
administered for treatment of a non-GVHD related condition and tapered to ≤ 0.5
mg/kg/day methylprednisolone (0.6 mg/kg/day prednisone) for seven or more days before
the onset of acute GVHD, the patient is eligible.

- Patients who are pregnant, breast feeding, or, if sexually active, unwilling to use
effective birth control for the duration of the study. Available evidence and/or
expert consensus is inconclusive or is inadequate for determining infant risk when
used during breastfeeding, therefore breast feeding patients are not eligible.

- Adults unable to provide informed consent.

- Patients on dialysis.

- Patients with severe hepatic VOD or sinusoidal obstruction syndrome who in the
judgement of the treating physician are not expected to have normalized bilirubin by
Day 56 after enrollment.

- Patients with a history of intolerance/allergy to MMF.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Outcome Measure:

GVHD-free survival

Outcome Time Frame:

Day 56

Safety Issue:


Principal Investigator

Javier Bolanos-Meade, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Johns Hopkins University


United States: Food and Drug Administration

Study ID:




Start Date:

January 2010

Completion Date:

June 2013

Related Keywords:

  • Graft-Versus-Host Disease
  • Immune System Disorders
  • GVHD
  • Graft vs Host Disease
  • Immune System Diseases



Johns Hopkins University Baltimore, Maryland  21205
Mayo Clinic Rochester, Minnesota  55905
Fred Hutchinson Cancer Research Center Seattle, Washington  98109
Memorial Sloan-Kettering Cancer Center New York, New York  10021
University of Iowa Hospitals and Clinics Iowa City, Iowa  52242
University of Pennsylvania Cancer Center Philadelphia, Pennsylvania  19104
Medical University of South Carolina Charleston, South Carolina  29425-0721
Medical College of Wisconsin Milwaukee, Wisconsin  53226
Rush University Medical Center Chicago, Illinois  60612-3824
City of Hope National Medical Center Los Angeles, California  91010
Baylor University Medical Center Dallas, Texas  75246
University of Minnesota Minneapolis, Minnesota  55455
Duke University Medical Center Durham, North Carolina  27710
University of Michigan Medical Center Ann Arbor, Michigan  48104-0914
Ohio State University Columbus, Ohio  43210
Texas Transplant Institute San Antonio, Texas  78229
Tufts Medical Center Boston, Massachusetts  02111
Oregon Health and Science University Portland, Oregon  97201
Virginia Commonwealth University Richmond, Virginia  
Stanford Hospital and Clinics Stanford, California  94305
Indiana BMT at Beech Grove Beech Grove, Indiana  46107
BMT Program at Northside Hospital Atlanta, Georgia  30342
Avera Hematology & Transplant Center Sioux Falls, South Dakota  57105
University of North Carolina Hospital at Chapel Hill Chapel Hill, North Carolina  27599
University of Florida College of Medicine Gainesville, Florida  32610
University of California San Diego Medical Center San Diego, California  92103-8409
Colorado Blood Cancer Institute Denver, Colorado  80218
DFCI, Massachusetts General Hospital Boston, Massachusetts  02114
Washington University, Barnes Jewish Hospital St. Louis, Missouri  63110
Weill Cornell Medical College, NY Presbyterian Hospital New York, New York  10065
University of Texas, MD Anderson Cancer Research Center Houston, Texas  77030
Levine Children's Hospital, Carolinas Medical Center Charlotte, North Carolina  28204
University of Maryland Medical Systems Baltimore, Maryland  21201
Dana Farber Cancer Institute, Brigham & Women's Hospital Boston, Massachusetts  02115
Hackensack Univ. Medical Center Hackensack, New Jersey  07601
Ann & Robert Lurie Children's Hospital of Chicago Chicago, Illinois  60611