Mismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome

Summary

This phase III trial compares hematopoietic (stem) cell transplantation (HCT) using mismatched related donors (haploidentical \[haplo\]) versus matched unrelated donors (MUD) in treating children, adolescents, and young adults with acute leukemia or myelodysplastic syndrome (MDS). HCT is considered standard of care treatment for patients with high-risk acute leukemia and MDS. In HCT, patients are given very high doses of chemotherapy and/or radiation therapy, which is intended to kill cancer cells that may be resistant to more standard doses of chemotherapy; unfortunately, this also destroys the normal cells in the bone marrow, including stem cells. After the treatment, patients must have a healthy supply of stem cells reintroduced or transplanted. The transplanted cells then reestablish the blood cell production process in the bone marrow. The healthy stem cells may come from the blood or bone marrow of a related or unrelated donor. If patients do not have a matched related donor, doctors do not know what the next best donor choice is. This trial may help researchers understand whether a haplo related donor or a MUD HCT for children with acute leukemia or MDS is better or if there is no difference at all.

Conditions

• Acute Lymphoblastic Leukemia
• Acute Myeloid Leukemia
• Mixed Phenotype Acute Leukemia
• Myelodysplastic Syndrome

Interventions

PROCEDURE

Haploidentical Hematopoietic Cell Transplantation

Undergo haploHCT

BIOLOGICAL

Lapine T-Lymphocyte Immune Globulin

Given IV

PROCEDURE

Lumbar Puncture

Undergo lumbar puncture

PROCEDURE

Matched Unrelated Donor Hematopoietic Cell Transplantation

Undergo MUD-HCT

DRUG

Melphalan

Given IV

DRUG

Methotrexate

Given IV

PROCEDURE

Multigated Acquisition Scan

Undergo MUGA

DRUG

Mycophenolate Mofetil

Given IV

PROCEDURE

Myeloablative Conditioning

Receive myeloablative conditioning regimen

OTHER

Quality-of-Life Assessment

Ancillary studies

OTHER

Questionnaire Administration

Ancillary studies

BIOLOGICAL

Rituximab

Given IV

PROCEDURE

T-Cell Depletion Therapy

Undergo haploHCT with alpha beta T cell depletion

DRUG

Tacrolimus

Given IV

DRUG

Thiotepa

Given IV

RADIATION

Total-Body Irradiation

Undergo TBI

DRUG

Busulfan

Given intravenously (IV)

DRUG

Cyclophosphamide

Given IV

PROCEDURE

Echocardiography Test

Undergo ECHO

DRUG

Fludarabine

Given IV

PROCEDURE

Biospecimen Collection

Undergo collection of blood

PROCEDURE

Bone Marrow Aspiration

Undergo bone marrow aspiration

Sponsors & Collaborators

• Children's Oncology Group

  lead NETWORK

Principal Investigators

• Heather J Symons · Children's Oncology Group

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

6 Months

Max Age

21 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-03-15

Primary Completion

2026-06-30

Completion

2026-06-30

FDA Drug

Yes

Countries

• United States
• Australia
• Canada

Study Locations