This is an academic open-label, phase II randomized study in patients with steroid resistant severe acute Graft versus host disease (GvHD) who have had allogeneic hematopoietic stem cell transplantation. The main purpose of this study is to compare the efficacy of Decidual Stromal Cells (DSC) with Investigators choice best available treatment (BAT). If randomized to DSC arm, patients will receive 2 infusions in the vein at least one week apart. Additional doses (up to 4 doses) of DSC may be given depending on response.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
50
Will vary depending upon Investigator's choice identified prior to randomization. Dose and frequency will depend on label (where approved) and institutional guidelines for various BAT. No cross-over to DSC treatment is planned. The BAT in this study will freely be identified by the Investigator prior to patient randomization and may include treatments such as: anti-thymocyte globulin (ATG), extracorporeal photopheresis (ECP), low-dose methotrexate (MTX), mTOR inhibitors (everolimus or sirolimus), vedolizumab, ruxolitinib.
The dose will be 1×106 DSC/kg bodyweight, at least 2 doses at least one week apart. Within the first 28 days, patients meeting criteria of aGvHD disease progression, mixed response or no response, may be given additional weekly doses of DSC until satisfactory response (ie: CR) are reached (max 4 doses in total).
Princess Margaret Cancer Centre
Toronto, Ontario, Canada
To compare the efficacy of DSC vs. Investigator's choice Best Available Therapy (BAT) in patients with Grade II-IV SR-aGvHD assessed by Durable Overall Response Rate (DOR) at Day 56
DSC vs. Investigator's choice Best Available Therapy (BAT) assessed by Durable Overall Response Rate (DOR) at Day 56
Time frame: Day 56
To assess Overall Response Rate at day 28 (ORR)
Overall Response Rate at day 28
Time frame: 28 days
To assess 1-year Overall Survival (OS)
1-year Overall Survival (OS)
Time frame: 1 year
To assess 1-year Non-Relapse Mortality (NRM)
1-year Non-Relapse Mortality (NRM)
Time frame: 1 year
To assess incidence of infections
number of infections
Time frame: up to 5 years, through study completion
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Adverse events reporting
Time frame: up to 5 years, through study completion
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