Phase 2 Study of Low-Dose Post-Transplant Cyclophosphamide to Prevent Graft-versus-Host Disease After Allogeneic Stem Cell Transplantation for Blood Cancers

What this Trial Is About

Researchers are evaluating the safety and effectiveness of a low-dose post-transplant cyclophosphamide PTCy regimen to prevent graft-versus-host disease GVHD in patients receiving allogeneic stem cell transplants for blood cancers. This Phase 2, single-arm study focuses on patients undergoing reduced-intensity or non-myeloablative conditioning and receiving stem cells from matched sibling, matched unrelated, or haploidentical donors. The trial aims to see if a lower dose of PTCy can reduce toxicity while maintaining similar outcomes compared to higher doses. Participants will receive 25 mgkg of PTCy on days 3 and 4 after their transplant, combined with tacrolimus and mycophenolate mofetil MMF for GVHD prevention. The study includes transplants from matched sibling, matched unrelated, single allelic mismatched unrelated, and haploidentical donors using peripheral blood stem cells. The conditioning regimens are either reduced-intensity or non-myeloablative. This approach is based on previous studies showing that lower PTCy doses may be effective with less toxicity. During the study, participants will be monitored for one year to assess outcomes such as survival without severe acute GVHD, chronic GVHD requiring treatment, and relapse or disease progression. Evaluations include regular clinical assessments of GVHD status and disease progression. The primary goal is to measure one-year GVHD-Free Relapse-Free Survival GRFS, with ongoing safety monitoring and follow-up through the study duration ending in 2031.

Allogeneic HSCT With Low-Dose Post-Transplant Cyclophosphamide for GVHD Prevention