Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Safety and Efficacy of Prochymal for the Salvage of Treatment-Refractory Acute GVHD Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Osiris Therapeutics
ClinicalTrials.gov Identifier:
NCT00284986
First received: January 31, 2006
Last updated: October 10, 2012
Last verified: October 2012
  Purpose

This study is designed to evaluate the safety and efficacy of Prochymal(TM) (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) in subjects experiencing treatment-refractory acute GVHD, Grades III-IV, that is refractory to standard first line therapies and at least one second-line therapy.


Condition Intervention Phase
Graft Versus Host Disease
Drug: Prochymal (TM)
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II Open Label Study to Evaluate the Safety and Efficacy of Prochymal (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) Infusion for the Salvage of Treatment-Refractory Acute GVHD Patients

Resource links provided by NLM:


Further study details as provided by Osiris Therapeutics:

Primary Outcome Measures:
  • Primary efficacy endpoint
  • - Response by Day 28

Secondary Outcome Measures:
  • Secondary efficacy endpoints
  • Improvement of GVHD by day 28 in one or more organs
  • involved with GVHD symptoms at day 1
  • Time to best overall GVHD grade
  • Best stage of each involved organ by day 28
  • Time to improvement or resolution of GVHD in one or
  • more organs
  • Safety Endpoints
  • Adverse events
  • Infusional toxicity
  • Overall relapse of underlying disease
  • Overall survival
  • Formation of ectopic tissue foci
  • Incidence of infection

Enrollment: 15
Study Start Date: November 2005
Study Completion Date: February 2007
Primary Completion Date: February 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Prochymal Drug: Prochymal (TM)

Detailed Description:

Allogeneic hematopoetic stem cell transplantation (HCT) is used in the treatment of a variety of hematological, myeloproliferative and lymphoproliferative disorders, and malignancies involving solid tumors. Patients receiving HCT can develop a life threatening condition called graft versus host disease (GVHD). GVHD occurs when donor T cells from the donor bone marrow recognize host cells as "foreign" and initiate an inflammatory immunological response. The standard of care for treatment of acute GVHD consists of intravenous delivery of methylprednisolone starting on day 1 and continuation of either cyclosporine or tacrolimus. This regimen of steroids and immunosuppressive drugs may relieve symptoms of GVHD, but some patients are refractory to current standard of care treatment. For treatment-refractory patients with grades III-IV GVHD mortality is approximately 80%. A therapy that could effectively suppress the immunological response from GVHD and help repair the damaged tissue could significantly decrease the mortality rate from this disease.

  Eligibility

Ages Eligible for Study:   6 Months to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects must be 6 months to 70 years of age inclusive.
  • If female and of childbearing age, subjects must be non-pregnant, not breast-feeding, and use adequate contraception. Male subjects must use adequate contraception.
  • Subjects must have Grade III-IV acute GVHD that has failed to respond to standard first and at least one second-line therapy. Biopsy for confirmation of both skin and gastrointestinal GVHD is not mandatory, but is recommended when feasible. Enrollment should not be delayed awaiting biopsy results.
  • Subjects must have minimal renal function as defined by:Calculated CrCl of > 30 mL/min using the Cockroft Gault equation.
  • Subject must provide written informed consent and authorization for use and disclosure of PHI.

Exclusion Criteria:

  • Subject has uncontrolled alcohol or substance abuse within 6 months of treatment.
  • 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 (e.g., uncontrolled infection, right heart failure, pulmonary hypertension, etc.).
  • Subject has a clinically significant, unstable arrhythmia.
  • Subject has a known allergy to bovine or porcine products.
  • Subject is unwilling to sign consent form for the long-term follow-up study, protocol No. 271
  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: NCT00284986

Locations
United States, North Carolina
Duke University
Durham, North Carolina, United States, 27708
Sponsors and Collaborators
Osiris Therapeutics
Investigators
Principal Investigator: Joanne Kurtzberg, M.D. Duke University
  More Information

No publications provided

Responsible Party: Osiris Therapeutics
ClinicalTrials.gov Identifier: NCT00284986     History of Changes
Other Study ID Numbers: Osiris 270
Study First Received: January 31, 2006
Last Updated: October 10, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Osiris Therapeutics:
GVHD
Graft vs Host Disease
Graft versus Host Disease

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

ClinicalTrials.gov processed this record on November 24, 2014