The study compares two acute graft-versus-host disease (aGVHD) prophylaxis regimens: efprezimod alfa vs placebo with the standard GVHD prophylaxis of tacrolimus / methotrexate. The study compares two acute graft-versus-host disease (aGVHD) prophylaxis regimens: efprezimod alfa/tacrolimus / methotrexate (efprezimod alfa/Tac/MTX) versus placebo/tacrolimus / methotrexate (placebo/Tac/MTX) in the setting of myeloablative conditioning (MAC), matched unrelated donor (MUD) allogeneic hematopoietic stem cell transplantation in participants with acute leukemia (AML/ALL) or myelodysplastic syndrome (MDS). The study agent, efprezimod alfa, will be administered through IV infusion on days -1, 14, and 28 at the dose of 480mg, 240 mg and 240mg, respectively. The placebo will be 100 ml normal saline intravenous (IV) solution.
The Sponsor decided to discontinue screening and enrollment in this study on 18 May 2021 for business reasons. This decision was not related to any new or unexpected safety or efficacy findings.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
11
IV infusion: 480 mg at Day -1, 240 mg at Day 14, 240 mg at Day 28.
IV infusion, 100 ml at Day -1, Day 14, and Day 28.
IV, 15 mg/m\^2/dose at Day 1, then 10 mg/m2/dose at Day 3, 6, 11.
Begin on day -3. IV \[0.03 mg/kg/day\] or PO \[0.045 mg/kg/dose\] dosing is permitted
City of Hope ( Site 0302)
Duarte, California, United States
The University of Chicago Medical Center ( Site 0306)
Chicago, Illinois, United States
Penn State University Milton S. Hershey Medical Center ( Site 0304)
Hershey, Pennsylvania, United States
Abramson Cancer Center of the University of Pennsylvania ( Site 0309)
Philadelphia, Pennsylvania, United States
180 Day Grade III-IV Acute Graft-Versus-Host Disease (GVHD)-Free Survival (aGFS)
Grade III-IV aGFS was defined as the time in days to the earlier of the first documented acute Grade III-IV GVHD or death by any cause in 180 days after hematopoietic stem cell transplantation (HCT). Event grading was based on the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v4.03 criteria. Participants who did not drop out or experience aGVHD or death were censored at the time of leukemia relapse or Day 180, whichever came first.
Time frame: Up to 180 days after HCT
Overall Survival (OS)
OS is defined as the time from HCT to death due to any cause. Participants who were alive at the end of the 180 days post HCT were censored at the last date they were known to be alive.
Time frame: Up to 180 days after HCT
Disease Free Survival (DFS)
DFS is defined as the earlier of time to leukemia relapse, or death, whichever occurred first. Subjects that were alive and did not experience disease relapse at the end of the follow-up period (180 days after HCT) were censored at the last date of evaluation.
Time frame: Up to 180 days after HCT
180 Day Grade II-IV aGFS
Grade II-IV aGFS was defined as the time in days to the earlier of the first documented acute Grade II-IV GVHD or death by any cause in 180 days after HCT. Event grading was based on the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v4.03 criteria. Participants who did not drop out or experience aGVHD or death were censored at the time of leukemia relapse or Day 180, whichever came first.
Time frame: Up to 180 days after HCT
180 Day Grade III-IV aGVHD-free and Relapse-free Survival (aGVHD RFS)
Grade III-IV aGVHD RFS was defined as the time in days to the earlier of the first documented acute Grade III-IV aGVHD, relapse, or death by any cause in 180 days after HCT. Event grading was based on the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v4.03 criteria. Participants who did not drop out, experience Grade III-IV aGVHD, relapse, or death were censored at Day 180.
Time frame: Up to 180 days after HCT
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