NCT06926595

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

Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) With Low-Dose Post-Transplant Cyclophosphamide for Prophylaxis of Graft-versus-Host Disease in Hematological Malignancies

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 41 (estimated)
• Sponsor: Milton S. Hershey Medical Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This study investigates the use of low-dose post-transplant cyclophosphamide (PTCy) for GVHD prophylaxis in a Phase 2, single-arm design. It involves patients receiving allogeneic stem cell transplantation with peripheral blood stem cells from matched sibling, matched unrelated, and haploidentical donors, following reduced-intensity or non-myeloablative conditioning. The aim is to evaluate 1-year GVHD-Free Relapse-Free Survival (GRFS) using 25 mg/kg of PTCy administered on days +3 and +4, in combination with tacrolimus and mycophenolate mofetil (MMF). The study builds on previous research that has shown the effectiveness of PTCy in preventing GVHD in various transplant settings, including haploidentical and matched unrelated donors. Lower doses of PTCy, such as 25 mg/kg, have been shown to be effective in other settings with reduced toxicity compared to the standard 50 mg/kg dose, and this study will assess whether the same holds true for peripheral blood stem cell transplants. The findings from this trial will help determine the optimal dosing strategy for PTCy in GVHD prophylaxis, with the potential to improve patient outcomes by minimizing toxicity while maintaining efficacy.

Conditions

• Graft-versus-Host Disease (GVHD)
• Hematologic Malignancies
• Allogeneic Stem Cell Transplantation

Interventions

Type	Name	Description
DRUG	Cyclophosphamide (primary intervention for GVHD prophylaxis)	This 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

Timeline

• Start date: 2026-03-09
• Primary completion: 2030-11-01
• Completion: 2031-11-01
• First posted: 2025-04-13
• Last updated: 2026-04-03

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study