drugset / Trial / NCT04904588

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

Phase 2 Active not recruiting 300 enrolled Center for International Blood and Marrow Transplant Research National Marrow Donor Program · collab

Non-randomizedParallel-groupOpen-labelTreatment

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.

Timeline

Start: 2021-09-30
Primary completion: 2026-08
Completion: 2026-12

Publications

Al Malki MM, Bo-Subait S, Logan B, Olson J, Kou J, Smith S, Leckrone E, Wu J, Stefanski HE, Auletta JJ, Spellman SR, Malmberg C, Askar M, Cusatis R, Shaffer BC, Modi D, Khimani F, Gooptu M, Hamadani M, Madbouly A, Maiers M, Fingerson S, Cook R, Ballen K, Loren A, Larkin K, Arai S, Qayed M, Choi SW, Broglie L, Shaw BE, Devine SM, Jimenez Jimenez AM. Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis After Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation. J Clin Oncol. 2025 Sep;43(25):2772-2781. doi: 10.1200/JCO-25-00856. Epub 2025 Jun 16.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Busulfan	Small molecule	6.4 mg/kg	Intravenous
Subject	Busulfan	Small molecule	8 mg/kg	Intravenous
Subject	Busulfan	Small molecule	9 mg/kg	Intravenous
Subject	Cyclophosphamide	Other / unclassified	29 mg/kg	Intravenous
Subject	Cyclophosphamide	Other / unclassified	50 mg/kg	Intravenous
Subject	Cyclophosphamide	Other / unclassified	100 mg/kg	Intravenous
Subject	Fludarabine	Small molecule	90 mg/m2	Intravenous
Subject	Fludarabine	Small molecule	120 mg/m2	Intravenous
Subject	Fludarabine	Small molecule	150 mg/m2	Intravenous
Subject	Fludarabine	Small molecule	180 mg/m2	Intravenous
Subject	Melphalan	Small molecule	100 mg/m2	Intravenous
Subject	Melphalan	Small molecule	140 mg/m2	Intravenous
Background	2-MERCAPTOETHANESULFONIC ACID	Small molecule	—	Intravenous
Background	Mycophenolate Mofetil	Small molecule	15 mg/kg	Intravenous
Background	Tacrolimus	Small molecule	0.03 mg/kg	Oral
Background	Tacrolimus	Small molecule	0.05 mg/kg	Oral

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

Summary

Timeline

Publications

Drugs

Indications