Treosulfan/Fludarabine/Low Dose TBI as a Preparative Regimen for Children With AML/MDS Undergoing Allo HCT
Status:
Completed
Trial end date:
2015-08-01
Target enrollment:
Participant gender:
Summary
This is a prospective, open-label, nonrandomized, prospective clinical trial evaluating a
fixed regimen of treosulfan, fludarabine and low-dose total body irradiation (TBI) in
children with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing
allogeneic hematopoietic cell transplantation (HCT). The primary hypothesis is that HCT with
a preparative regimen consisting of treosulfan, fludarabine and low-dose TBI will result in
overall survival (OS) comparable to historical rates observed with conventional myeloablative
regimens in the pediatric population. The preparative regimen will result in adequate
incidence of neutrophil and platelet engraftment, and acceptable rates of graft-versus-host
disease (GVHD), relapse and survival. The pharmacokinetic (PK) profile of treosulfan in
children will be comparable to that of adults previously studied.
Phase:
Phase 2
Details
Lead Sponsor:
Center for International Blood and Marrow Transplant Research
Collaborators:
medac GmbH National Marrow Donor Program Pediatric Blood and Marrow Transplant Consortium Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI BMT) St. Baldrick's Foundation