NCT04904588

HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide

A Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide for Patients With Hematologic Malignancies

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 300 (estimated)
• Sponsor: Center for International Blood and Marrow Transplant Research · Network
• Sex: All
• Age: 1 Year
• Healthy volunteers: Not accepted

Summary

This is a prospective, multi-center, Phase II study of hematopoietic cell transplantation (HCT) using human leukocyte antigen (HLA)-mismatched unrelated donors (MMUD) for peripheral blood stem cell transplant in adults and bone marrow stem cell transplant in children. Post-transplant cyclophosphamide (PTCy), tacrolimus and mycophenolate mofetil (MMF) will be used for for graft versus host disease (GVHD) prophylaxis. This trial will study how well this treatment works in patients with hematologic malignancies.

Conditions

• Acute Lymphoblastic Leukemia
• Acute Myelogenous Leukemia
• Mixed Phenotype Acute Leukemia
• Acute Leukemia
• Myelodysplastic Syndromes
• Chronic Myelogenous Leukemia
• Chronic Lymphocytic Leukemia
• Lymphoma

Interventions

Type	Name	Description
DRUG	Busulfan	Given IV or PO pre-transplant as part of conditioning regimen
DRUG	Busulfan	Given IV pre-transplant as part of conditioning regimen
DRUG	Fludarabine	Given IV pre-transplant as part of conditioning regimen
RADIATION	Total-body irradiation	Administered pre-transplant as part of conditioning regimen
DRUG	Cyclophosphamide	Given IV pre-transplant as part of conditioning regimen
DRUG	Melphalan	Given IV pre-transplant as part of conditioning regimen
PROCEDURE	PBSC Hematopoietic Stem Cell Transplantation (HSCT)	Peripheral blood stem cell graft is infused from a mismatched unrelated donor on Day 0.
PROCEDURE	Bone Marrow Hematopoietic Stem Cell Transplantation	Bone marrow graft is infused from a mismatched unrelated donor on Day 0.
DRUG	Post-transplant Cyclophosphamide	Cyclophosphamide (50 mg/kg) is administered on Day +3 and on Day +4 post-transplant as an IV infusion over 1-2 hours.
DRUG	Mesna	Mesna is given in divided doses IV 30 min pre- and at 3, 6, and 8 hours post-cyclophosphamide.
DRUG	Tacrolimus	Tacrolimus is given at a dose of 0.05 mg/kg PO or an IV dose of 0.03 mg/kg of ideal body weight (IBW) starting on Day +5 post-transplant with taper recommended at 90-100 days post HCT.
DRUG	Mycophenolate Mofetil	Mycophenolate mofetil (MMF) is given at a dose of 15 mg/kg three times daily IV or PO from Day +5 to Day +35 post-transplant.
OTHER	Patient-Reported Outcomes	Survey assessments will be administered to study participants pre- and post-transplant.

Timeline

• Start date: 2021-09-30
• Primary completion: 2026-08-01
• Completion: 2026-12-01
• First posted: 2021-05-27
• Last updated: 2026-03-18

Locations

39 sites across 1 country: United States