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A Phase III, Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Prochymal(Ex Vivo Cultured Adult Human Mesenchymal Stem Cells) Infusion for the Treatment of Steroid Refractory Acute GVHD


Phase 3
6 Months
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(Ex Vivo Cultured Adult Human Mesenchymal Stem Cells) Infusion for the Treatment of Steroid Refractory Acute GVHD


Approximately 6300 patients receive allogeneic hematopoietic stem cell transplants in the
United States each year (IBMTR, 2003). Nearly 50% (approximately 3,150) of these patients
develop acute GVHD (Goker et al). A fraction of these patients (approximately 870) will
progress to the severe stages of the disease, Grades III-IV. It is estimated that nearly 82%
of those patients with severe acute GVHD will be steroid refractory (Przepiorka et al.,
1995) and of these, only 50% of steroid-refractory patients wll respond to secondary and
tertiary treatments (Greinix et al., 2000). Thus, roughly 350 patients each year face
tremendous odds against survival. In addition, most patients who initially responded to
secondary and tertiary treatments have a high risk of dying within the first year (Remberger
et al., 2001; Anasetti et al., 1994). Development of new therapeutic agents and strategies
to rescue patients with steroid refractory, acute GVHD would provide a significant benefit
in an area of unmet medical need.

Patients will receive standard of care in addition to adult mesenchymal stem cells or
placebo.


Inclusion Criteria:



- Subjects must be 6 months to 70 years of age, inclusive

- Subjects who have failed to respond to steroid treatment: Failure to respond to
steroid treatment is defined as any grade B-D (IBMTR grading) of acute GVHD that
shows: No improvement after 3 days and a duration of no greater than 2 weeks while
receiving treatment with Methylprednisolone (greater than or equal to 1 mg/kg/day) or
equivalent.

- Subjects must be treated within 4 days of randomization . In urgent situations 2nd
line therapy may be started 24 hours prior to randomization , and Prochymal must be
initiated within the following 3 days.

- Subjects who have received an increase in their steroid dose treatment prior to
randomization will be eligible for enrollment. An increase in steroid dose will not
be considered as second line therapy.

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

- For pediatric patients: Schwartz equation: (Patient population: infants over 1 week
old through adolescence (<18 years old)

- 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 started treatment with second line therapy >24 hours prior to
randomization.

- Subject has received agents other than steroids for primary treatment of acute GVHD

- Subject is participating in the CTN Protocol 0302

- 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)
concurrently during study participation or within 30 days of randomization.

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

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

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

Complete Response of greater than or equal to 28 days duration

Outcome Time Frame:

Day 100

Safety Issue:

No

Principal Investigator

Paul Martin, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Fred Hutchinson Cancer Center

Authority:

United States: Food and Drug Administration

Study ID:

280

NCT ID:

NCT00366145

Start Date:

July 2006

Completion Date:

May 2009

Related Keywords:

  • Graft Versus Host Disease
  • acute GVHD
  • steroid refractory GVHD
  • severe steroid refractory acute GVHD
  • steroid refractory
  • Steroid Refractory Acute Graft Versus Host Disease
  • Graft vs Host Disease

Name

Location

Louisiana State UniversityNew Orleans, Louisiana  70112-2282
Arizona Cancer CenterTucson, Arizona  85724
MD Anderson Cancer CenterHouston, Texas  77030-4096
Fred Hutchinson Cancer Research CenterSeattle, Washington  98109
Medical University of South CarolinaCharleston, South Carolina  29425-0721
Beth Israel Deaconess Medical CenterBoston, Massachusetts  02215
Medical College of WisconsinMilwaukee, Wisconsin  53226
New York Medical CollegeValhalla, New York  10595
Rush University Medical CenterChicago, Illinois  60612-3824
Dana-Farber Cancer InstituteBoston, Massachusetts  02115
University of Kansas Medical CenterKansas City, Kansas  66160-7353
Hackensack University Medical CenterHackensack, New Jersey  07601
All Children's HospitalSt. Petersburg, Florida  33701
Mount Sinai Medical CenterNew York, New York  10029
Western Pennsylvania Cancer InstitutePittsburgh, Pennsylvania  15224
City of HopeDuarte, California  91010
Duke UniversityDurham, North Carolina  27710
University of Alabama at BirminghamBirmingham, Alabama  35294-3300
Baylor UniversityWaco, Texas  
University of RochesterRochester, New York  14642
Emory UniversityAtlanta, Georgia  30322
Northwestern Center for Clinical ResearchChicago, Illinois  60611
University of LouisvilleLouisville, Kentucky  40202
University of MiamiMiami, Florida  33136
Oregon Health and Science UniversityPortland, Oregon  97201
New York Presbyterian HospitalNew York, New York  10021
Texas Cancer Center at Medical CityDallas, Texas  75230
Univeristy of Texas Southwestern Medical CenterDallas, Texas  
University of NebraskaOmaha, Nebraska  68198
Penn State Hershey Medical CenterHershey, Pennsylvania  17033
Northside HospitalAtlanta, Georgia  30342
Mayo Clinic RochesterRochester, Minnesota  55905
Tufts-New England Medical CenterBoston, Massachusetts  02111
Univeristy of California San FranciscoSan Francisco, California  94143
Yale New Haven HospitalNew Haven, Connecticut  06520
University of Illinois - ChicagoChicago, Illinois  60612
Indiana Blood and Bone Marrow Transplant CenterBeech Grove, Indiana  46107
Univeristy of Iowa Hospitals and ClinicsIowa City, Iowa  52242
University of Maryland/GreenbaumBaltimore, Maryland  21201
Karmanos/Wayne State UniversityDetroit, Michigan  48201
Univeristy of Mississippi Medical CenterJackson, Mississippi  39216
Roswell ParkBuffalo, New York  14263
Columbia University/New York Presbyterian HospitalNew York, New York  10032
Wake Forest Univeristy School of MedicineWinston Salem, North Carolina  27157
Texas Research CenterSan Antonio, Texas  78229
Virginia Commonwealth/Massey Cancer CenterRichmond, Virginia  23298
University of Wisconsin MadisonMadison, Wisconsin  53792