This Phase 2, single-arm, open-label study aims to evaluate the safety and efficacy of low-dose (25 mg/kg) post-transplant cyclophosphamide (PTCy) for prophylaxis of Graft-versus-Host Disease (GVHD) in patients undergoing allogeneic stem cell transplantation following reduced-intensity or non-myeloablative conditioning. The study will focus on matched sibling, matched unrelated, and haploidentical peripheral blood stem cell donors. The primary endpoint is 1-year GVHD-Free Relapse-Free Survival (GRFS). The study seeks to determine if low-dose PTCy offers similar outcomes as higher doses, with potentially reduced toxicity.
Age 18 or older at the time of study enrollment.
Patients with acute leukemia (acute myeloid leukemia, acute lymphoblastic leukemia, mixed phenotype acute leukemia) or chronic myeloid leukemia with no circulating blasts and less than 5% blasts in the bone marrow.
Patients with myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia with no circulating blasts and less than 10% blasts in the bone marrow (exception allowed due to lack of difference in outcomes with <5% vs 5-10% blasts in this disease).
Patients with secondary acute myeloid leukemia progressing from pre-existing myelodysplastic syndrome, myeloproliferative disease (MPN), or MDS/MPN overlap syndrome.
Prior allogeneic stem cell transplant.
Active central nervous system (CNS) involvement by malignant cells.
Uncontrolled bacterial, viral, or fungal infections (currently taking medication with progression or no clinical improvement).
Seropositive for human immunodeficiency virus (HIV) with detectable viral load, hepatitis B virus (HBV) or hepatitis C virus (HCV) with detectable viral load. Hepatitis B surface antibody positive due to vaccination or natural immunity are permitted. Patients previously treated for HCV and considered to be in sustained virologic remission (SVR) are allowed.
Single group
Cyclophosphamide (primary intervention for GVHD prophylaxis)
DrugThis study evaluates the use of low-dose (25 mg/kg) post-transplant cyclophosphamide (PTCy) for prophylaxis of Graft-versus-Host Disease (GVHD) following allogeneic stem cell transplantation. The intervention involves administering PTCy on days +3 and +4 post-transplant, in combination with tacrolimus and mycophenolate mofetil (MMF) for GVHD prevention. The goal is to assess the safety and efficacy of this regimen in patients undergoing reduced-intensity or non-myeloablative conditioning using peripheral blood stem cells from matched sibling, matched unrelated, and haploidentical donors
Number of Subjects Without Grade III/IV Acute Graft-Versus-Host Disease (GVHD) at 1 Year
The number of subjects without Grade III/IV acute graft-versus-host disease (GVHD) at one year following allogeneic stem cell transplantation, assessed using the Modified Glucksberg Criteria (Grade III: severe organ involvement, e.g., skin stage 3-4, liver or gut stage 2-3; Grade IV: life-threatening dysfunction, e.g., skin, liver, or gut stage 4).
Number of Subjects Without Chronic Graft-Versus-Host Disease (GVHD) Requiring Systemic Treatment at 1 Year
The number of subjects without chronic graft-versus-host disease (GVHD) requiring systemic treatment (e.g., corticosteroids or immunosuppressive therapy) at one year following allogeneic stem cell transplantation with low-dose post-transplant cyclophosphamide (25 mg/kg), assessed per the NIH Consensus Criteria for chronic GVHD.
Number of Subjects Without Relapse or Disease Progression at 1 Year
The number of subjects without relapse or disease progression at one year following allogeneic stem cell transplantation, defined as the reappearance of the underlying disease or worsening of disease burden, assessed by standard clinical and laboratory measures (e.g., bone marrow biopsy, or disease-specific markers such as minimal residual disease \[MRD\] testing) per the study protocol.
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