This early phase I trial is to find out the effect of adding cord blood tissue-derived mesenchymal stromal cells (cb-MSCs) to ruxolitinib in treating patients with acute graft versus host disease that does not respond to steroid therapy (steroid-refractory). Ruxolitinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. cb-MSCs are a type of tissue helper cell that can be removed from donated umbilical cord blood tissue and grown into many different cell types that can be used to treat cancer and other disease, such as graft versus host disease. This trial aims to learn if adding cb-MSCs to ruxolitinib may help control steroid-refractory acute graft versus host disease.
Participants between the ages of 12 years and 80 years (inclusive).
Steroid refractory grades II-IV acute GVHD of the Lower GI tract or Liver (including those developing these manifestations after previous acute GVHD of skin) secondary to allogeneic HCT or donor lymphocyte infusion. (Grading, see Appendix I) GVHD with: No improvement after treatment with methylprednisolone at ≥ 2.0 mg/kg/day or equivalent for minimum 7 days, or progressive symptoms after minimum 3 days, or a flare in acute GVHD while on systemic steroids. Participants must have had a biopsy that suggests GVHD; a repeat biopsy to enroll on the study is not necessary.
Karnofsky/Lansky Performance score of at least 30 at the time of study entry.
Participants who are women of childbearing potential, must be non-pregnant, not breast-feeding, and use adequate contraception. Male patients must use adequate contraception
De novo chronic GVHD
Isolated acute GVHD of skin
Secondary systemic therapy for acute GVHD ruxolitinib greater than 96 hours before initiation of therapy.
Primary treatment with agents other than alpha-1 antitrypsin (AAT) glucocorticoids and ruxolitinib.
Parallel
Cellular Therapy
Other interventionGiven ds-MSCs IV
Ruxolitinib
DrugGiven PO
Death from any cause
Response
Will compare the patient's 28-day graft versus host disease (GVHD) status to the patient's baseline GVHD status when steroid refractory acute GVHD was diagnosed.
Incidence of adverse events
Graft versus host disease status
Will assess complete response (CR), very good partial response (VGPR), partial response (PR), stable disease (SD), and progressive disease (PD).
Proportion of response
Will assess proportion of patients with CR, PR, VGPR, and no-response (NR). The event NR is defined as stable disease, mixed response, disease progression, OR initiation of additional systemic (second-line) GVHD therapies.
Time to complete response
Will be estimated by the method of Kaplan and Meier.
Time to very good partial response
Will be estimated by the method of Kaplan and Meier.
Cytokine biomarker analysis (optional)
Will use cytokine biomarker assays to predict response to therapy.
Fecal samples analysis (optional)
Will use fecal samples to assess microbiome and potential predictor for response to therapy.
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M.D. Anderson Cancer Center
Lead sponsor
National Cancer Institute (NCI)
Collaborator
