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Efficacy and Safety of Adult Human Mesenchymal Stem Cells to Treat Steroid Refractory Acute Graft Versus Host Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Osiris Therapeutics
ClinicalTrials.gov Identifier:
NCT00366145
First received: August 17, 2006
Last updated: October 11, 2011
Last verified: October 2011
History of Changes
  Purpose
The purpose of this study is to evaluate the efficacy and gather additional safety of Prochymal in subjects who have failed to respond to steroid treatment of Grades B-D acute GVHD.

Condition Intervention Phase
Graft Versus Host Disease Biological: Mesenchymal Stem Cells Biological: placebo Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Osiris Therapeutics:

Primary Outcome Measures:
  • Complete Response of greater than or equal to 28 days duration [ Time Frame: Day 100 ]

Secondary Outcome Measures:
  • Survival at 180 days post first infusion [ Time Frame: 180 Days ]
Enrollment: 240
Study Start Date: July 2006
Study Completion Date: May 2009
Primary Completion Date: May 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 1
Patients who receive standard of care plus treatment with ex vivo cultured adult human Mesenchymal Stem Cells
 Biological: Mesenchymal Stem Cells
2 million cells/kg twice a week for 4 weeks
Other Name: Prochymal
Placebo Comparator: 2
Patients who receive standard of care and do not receive treatment with ex vivo cultured adult human mesenchymal stem cells.
 Biological: placebo
2 infusions a week for 4 weeks
Other Name: excipients without adult human mesenchymal stem cells

Detailed Description:

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.

  Eligibility
Ages Eligible for Study:   6 Months to 70 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00366145

  Show 70 Study Locations
Sponsors and Collaborators
Osiris Therapeutics
Investigators
Principal Investigator: Paul Martin, MD Fred Hutchinson Cancer Center
  More Information

Additional Information:
Click here for more information about this study: A Phase III 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  This link exits the ClinicalTrials.gov site

Publications:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Osiris Therapeutics
ClinicalTrials.gov Identifier: NCT00366145     History of Changes
Obsolete Identifiers: NCT00476840
Other Study ID Numbers: 280
Study First Received: August 17, 2006
Last Updated: October 11, 2011

Keywords provided by Osiris Therapeutics:
acute GVHD
steroid refractory GVHD
severe steroid refractory acute GVHD
steroid refractory
Steroid Refractory Acute Graft Versus Host Disease

Additional relevant MeSH terms:
Graft vs Host Disease
Immune System Diseases

ClinicalTrials.gov processed this record on June 23, 2017