NCT07249346

Dose-Expansion Study of Low Dose Post-Transplant Cyclophosphamide/Tacrolimus/Ruxolitinib for Graft-versus-Host Disease (GVHD) Prophylaxis in Myeloablative Allogeneic Peripheral Blood Stem Cell Transplantation

An Open Label, Non-Randomized, Multi-Center Pilot Dose-Expansion Study of Low Dose Post-Transplant Cyclophosphamide/Tacrolimus/Ruxolitinib for GVHD Prophylaxis in Myeloablative Allogeneic Peripheral Blood Stem Cell Transplantation

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 124 (estimated)
• Sponsor: Hannah Choe, MD · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is an open label, non-randomized, multicenter, pilot, dose expansion study of low dose post-transplant cyclophosphamide (25 mg/kg on Days +3 and +4)/tacrolimus/ruxolitinib in the setting of myeloablative conditioning (MAC) allogeneic peripheral blood stem cell transplantation (PBSCT).

Detailed description

Primary Objective: To assess survival without severe Grade 3-4 acute GVHD at Day 180 post-transplant in patients treated with GVHD prophylaxis in myeloablative allogeneic hematopoietic stem cell transplantation for patients treated with low dose PTCy(Cyclophosphamide)/Tac(Tacrolimus)/Rux(Ruxolitinib). Secondary Objectives: To describe rates of Grade II-IV and Grades III-IV acute GVHD, chronic GVHD requiring immunosuppression, hematologic recovery (neutrophil and platelet), disease relapse or progression, rates of Grade 3+ toxicity, primary and secondary graft failure, and overall survival (OS) for patients treated with low dose PTCy/Tac/Rux.

Conditions

• Leukemia
• Myelodysplasia
• Chronic Myelomonocytic Leukemia

Interventions

Type	Name	Description
DRUG	Ruxolitinib	Taken PO
DRUG	Myeloablative conditioning regimen	Patients will receive a full-intensity myeloablative conditioning regimen. Allowed regimens include: * Flu/Bu(130 mg/m2/day x 4 days) * Flu/TBI (8-12Gy) * Flu/Bu/Thiotepa The addition of alemtuzumab or ATG is not allowed.
PROCEDURE	Hematopoietic Stem Cell Transplantation	Patients will undergo HCT
DRUG	Cyclophosphamide	Given IV
DRUG	Tacrolimus	Given PO

Timeline

• Start date: 2026-06-01
• Primary completion: 2027-06-01
• Completion: 2027-06-01
• First posted: 2025-11-25
• Last updated: 2025-12-24

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study