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A Phase III, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Prochymal™ Infusion in Combination With Corticosteroids for the Treatment of Newly Diagnosed Acute GVHD


Phase 3
18 Years
70 Years
Not Enrolling
Both
Graft Versus Host Disease

Thank you

Trial Information

A Phase III, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Prochymal™ Infusion in Combination With Corticosteroids for the Treatment of Newly Diagnosed Acute GVHD


Subjects will be treated with a total of 6 infusions of investigational agent during the
first 4 weeks of the study. Four infusions will be administered during the first two weeks
(twice weekly), then two infusions administered during the next two weeks (once weekly).
Subjects assigned to the active treatment group will receive Prochymal™. Subjects assigned
to the non active treatment group will receive placebo (excipient, less cells). It is
recommended that all subjects receive all six infusions. The discontinuation of
investigational agent is allowed for GVHD worsening with subsequent need for salvage
therapy. All infusions must be given at least 3 days apart.

Subjects will be evaluated for efficacy and safety until death, withdrawal or 90 study days
after randomization, whichever occurs first. Study will be unblinded and data analyzed at
Day 90 post 1st infusion (Day 0)following final subject enrollment. Subjects will be
followed for safety for 12 months post 1st infusion (Day 0).


Inclusion Criteria:



- Subjects must be 18 years to 70 years of age, inclusive

- Subjects must have received an allogeneic hematopoietic stem cell transplant using
either bone marrow, peripheral blood stem cells or cord blood or administered a donor
leukocyte infusion.

- Subjects must have newly diagnosed Grades B-D acute GVHD. Biopsy confirmation of GVHD
is strongly recommended but not required. Randomization should not be delayed
awaiting biopsy or pathology results.

- Subjects must be randomized and treated with corticosteroid (1-2 mg/kg/d
methylprednisolone, or equivalent) and Prochymal™/placebo within 72 hours of onset of
acute GVHD.

- Subjects must have adequate renal function as defined by: Calculated Creatinine
Clearance of >30mL/min using the Cockroft-Gault equation

- Subjects who are women of childbearing potential, must be non-pregnant, not
breast-feeding, and use adequate contraception. Male subjects must use adequate
contraception

- Subject must have a minimum Karnofsky Performance Level of at least 30 at the time of
study entry

- Subject (or legal representative where appropriate) must be capable of providing
written informed consent.

Exclusion Criteria:

- Subject has been previously treated with systemic immunosuppressive therapy for acute
GVHD

- Subject has any underlying or current medical or psychiatric condition that, in the
opinion of the Investigator, would interfere with the evaluation of the subject
including uncontrolled infection, heart failure, pulmonary hypertension, etc.

- Subjects may not receive any other investigational agents (not approved by the FDA
for any indication) concurrently during study participation or within 30 days of
randomization.

- Subject has a known allergy to bovine or porcine products or DMSO

- Subject has received a transplant for a solid tumor disease.

- Subject requires more than 2L/min of oxygen to maintain stable SaO2 greater than or
equal to 92%

- Subject requires a renal dopamine dose greater than 1-3 mcg/kg/min to maintain renal
blood flow associated with renal failure and improved urinary output.

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

Achieved an induction of a complete response, Followed by 28 days of maintenance of a clinically meaningful response that does not did not require an increase in corticosteroid dose , did not require second line therapy and survived 90 days.

Outcome Time Frame:

90 Days

Safety Issue:

No

Principal Investigator

Rod L Monroy, Ph.D.

Investigator Role:

Study Director

Investigator Affiliation:

Osiris Therapeutics, Inc.

Authority:

United States: Food and Drug Administration

Study ID:

265

NCT ID:

NCT00562497

Start Date:

September 2007

Completion Date:

May 2010

Related Keywords:

  • Graft Versus Host Disease
  • Acute GVHD
  • Acute Graft Versus Host Disease
  • Graft vs Host Disease

Name

Location

Memorial Sloan Kettering Cancer Center New York, New York  10021
MD Anderson Cancer Center Houston, Texas  77030-4096
Roswell Park Cancer Institute Buffalo, New York  14263
Fred Hutchinson Cancer Research Center Seattle, Washington  98109
Indiana University Cancer Center Indianapolis, Indiana  46202-5265
Barbara Ann Karmanos Cancer Institute Detroit, Michigan  48201
University of Mississippi Medical Center Jackson, Mississippi  39216-4505
Washington University School of Medicine Saint Louis, Missouri  63110
Mount Sinai School of Medicine New York, New York  10029
Beth Israel Deaconess Medical Center Boston, Massachusetts  02215
Loyola University Medical Center Maywood, Illinois  60153
Medical College of Wisconsin Milwaukee, Wisconsin  53226
Western Pennsylvania Hospital Pittsburgh, Pennsylvania  15224
Rush University Medical Center Chicago, Illinois  60612-3824
Rocky Mountain Cancer Center Denver, Colorado  80218
Baylor University Medical Center Dallas, Texas  75246
Massachusetts General Hospital Boston, Massachusetts  02114-2617
University of Florida Gainesville, Florida  32610-0277
Tufts New England Medical Center Boston, Massachusetts  02111
Emory University Atlanta, Georgia  30322
Northwestern Center for Clinical Research Chicago, Illinois  60611
UCLA Medical Center Los Angeles, California  90095-7059
Thomas Jefferson University Philadelphia, Pennsylvania  19107-6541
Dana Farber Cancer Institute Boston, Massachusetts  02115
Ohio State University Columbus, Ohio  43210
University of Louisville Louisville, Kentucky  40202
Texas Transplant Institute San Antonio, Texas  78229
New York Presbyterian Hospital New York, New York  10021
Virginia Commonwealth University Richmond, Virginia  
Kansas City Cancer Center Kansas City, Missouri  64111
Jewish Hospital Cincinnati, Ohio  45236
Wake Forest University School of Medicine Winston-Salem, North Carolina  27157-1023
University of North Carolina Hospitals Chapel Hill, North Carolina  27599
Northside Hospital Atlanta, Georgia  30342
Penn State Milton S. Hershey Medical Center Hershey, Pennsylvania  17033
University of Chicago Hospitals Chicago, Illinois  60637
University of California Medical Center San Francisco, California  94143
University of Alabama Birmingham (UAB) Hospital Birmingham, Alabama  35249
St. Francis Cancer Center Indianapolis, Indiana  46237
Mayo Medical Center Rochester, Minnesota  55905
Duke University Health System Durham, North Carolina  27705
Abramson Cancer Center, University of Pennsylvania Health System Philadelphia, Pennsylvania  19104
University of Pittsburgh Cancer Centers Pittsburgh, Pennsylvania  15232
Oncology Hematology Association Pittsburgh, Pennsylvania  15232
Medical University of South Carolina(MUSC) Charleston, South Carolina  29425