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Treosulfan/Fludarabine/Low Dose TBI as a Preparative Regimen for Children With AML/MDS Undergoing Allo HCT

NCT01772953 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2016-10-12

No results posted yet for this study

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.

Conditions

  • Acute Myeloid Leukemia (AML)
  • Myelodysplastic Syndrome (MDS)

Interventions

DRUG

Treosulfan

This is a phase II, open-label, nonrandomized, prospective study of a preparative regimen consisting of treosulfan, fludarabine and low-dose total body irradiation (TBI) for children with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT).

Sponsors & Collaborators

  • National Marrow Donor Program

    collaborator OTHER

  • Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI BMT)

    collaborator UNKNOWN

  • Pediatric Blood and Marrow Transplant Consortium

    collaborator OTHER

  • St. Baldrick's Foundation

    collaborator OTHER

  • medac GmbH

    collaborator INDUSTRY

  • Center for International Blood and Marrow Transplant Research

    lead NETWORK

Principal Investigators

  • Eneida Nemecek, MD · Doernbecher Children's Hospital, Oregon Health & Science University

  • Colleen Delaney, MD · Seattle Children's Hospital, Fred Hutchinson Cancer Research Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-09-30
Primary Completion
2015-08-31
Completion
2015-08-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01772953 on ClinicalTrials.gov