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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
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

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

- 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

- 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

- 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:


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:


Principal Investigator

Paul Martin, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Fred Hutchinson Cancer Center


United States: Food and Drug Administration

Study ID:




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



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