This study is designed to evaluate the safety and efficacy of Prochymal® (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) in participants experiencing treatment-refractory acute GVHD, Grades III-IV, that is refractory to standard first-line therapies and at least one second-line therapy.
Allogeneic HSCT is used in the treatment of a variety of hematological, myeloproliferative and lymphoproliferative disorders, and malignancies involving solid tumors. Participants receiving HSCT can develop a life-threatening condition called 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 participants are refractory to current standard of care treatment. For treatment-refractory participants 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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
11
Intravenous infusion of ex-vivo cultured adult human mesenchymal stem cells
Duke University
Durham, North Carolina, United States
Response by Day 28
Responses included complete response (CR), partial response (PR), failure to respond. CR is defined as a complete resolution of graft-versus-host disease (GVHD). PR is defined as improvement in at least one organ by at least one full stage in the absence of progression in any other organ, or resolution of GVHD in at least one organ with a need for additional treatment because of abnormalities persisting in another organ. Failure to respond is defined as progression of GVHD.
Time frame: Day 28
Improvement of GVHD by Day 28 in one or more organs involved with GVHD symptoms at day 1
Time frame: Day 1
Best stage of each involved organ by Day 28
Time frame: Day 28
Time to improvement or resolution of GVHD in one or more organs
Time frame: Up to approximately 12 months
Adverse events
Time frame: Up to approximately 12 months
Infusional toxicity
Time frame: Up to approximately 12 months
Overall relapse of underlying disease
Time frame: Up to approximately 12 months
Overall survival
Time frame: Up to approximately 12 months
Formation of ectopic tissue foci
Time frame: Up to approximately 12 months
Incidence of infection
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Time frame: Up to approximately 12 months