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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00284986
Recruitment Status : Completed
First Posted : February 1, 2006
Last Update Posted : March 10, 2020
Information provided by (Responsible Party):
Mesoblast, Ltd. ( Mesoblast, Inc. )

Brief Summary:
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 or disease Intervention/treatment Phase
Graft Versus Host Disease Drug: Prochymal (TM) Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (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
Actual Study Start Date : November 18, 2005
Actual Primary Completion Date : April 25, 2006
Actual Study Completion Date : February 8, 2007

Arm Intervention/treatment
Experimental: PROCHYMAL™
Drug: Prochymal (TM)
Intravenous infusion of ex vivo cultured adult human mesenchymal stem cells

Primary Outcome Measures :
  1. Response by Day 28 [ Time Frame: Day 28 ]

Secondary Outcome Measures :
  1. Improvement of GVHD by Day 28 in one or more organs involved with GVHD symptoms at day 1 [ Time Frame: Day 1 ]
  2. Best stage of each involved organ by Day 28 [ Time Frame: Day 28 ]
  3. Time to improvement or resolution of GVHD in one or more organs [ Time Frame: Up to approximately 12 months ]
  4. Adverse events [ Time Frame: Up to approximately 12 months ]
  5. Infusional toxicity [ Time Frame: Up to approximately 12 months ]
  6. Overall relapse of underlying disease [ Time Frame: Up to approximately 12 months ]
  7. Overall survival [ Time Frame: Up to approximately 12 months ]
  8. Formation of ectopic tissue foci [ Time Frame: Up to approximately 12 months ]
  9. Incidence of infection [ Time Frame: Up to approximately 12 months ]

Information from the National Library of Medicine

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

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 creatinine clearance (CrCl) of > 30 mL/min using the Cockcroft-Gault equation.
  • Subject must provide written informed consent and authorization for use and disclosure of protected health information (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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00284986

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United States, North Carolina
Duke University
Durham, North Carolina, United States, 27708
Sponsors and Collaborators
Mesoblast, Inc.
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Study Director: Mahboob Rahman, MD Mesoblast, Inc.
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Responsible Party: Mesoblast, Inc. Identifier: NCT00284986    
Other Study ID Numbers: Osiris 270-271
First Posted: February 1, 2006    Key Record Dates
Last Update Posted: March 10, 2020
Last Verified: March 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Mesoblast, Ltd. ( Mesoblast, Inc. ):
Graft vs Host Disease
Graft versus Host Disease
Additional relevant MeSH terms:
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Graft vs Host Disease
Immune System Diseases