Mismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome (NCT05457556)
A Multi-Center, Phase 3, Randomized Trial of Matched Unrelated Donor (MUD) Versus HLA-Haploidentical Related (Haplo) Myeloablative Hematopoietic Cell Transplantation for Children, Adolescents, and Young Adults (AYA) With Acute Leukemia or Myelodysplastic Syndrome (MDS)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (73 items) and avoid the listed exclusions (11 items).
Location: Children's Hospital of Alabama — Birmingham, Alabama
Age range: 6 Months to 21 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion Criteria:
- PATIENT EXCLUSION CRITERIA FOR ENROLLMENT:
- Patients with genetic disorders (generally marrow failure syndromes) prone to secondary AML/ALL/MPAL with known poor outcomes because of sensitivity to alkylator therapy and/or TBI are not eligible (Fanconi Anemia, Kostmann Syndrome, Dyskeratosis Congenita, etc). Patients with Downs syndrome because of increased toxicity with intensive conditioning regimens.
- Patients with any obvious contraindication to myeloablative HCT at the time of enrollment
- Female patients who are pregnant are ineligible as many of the medications used in this protocol could be harmful to unborn children and infants
- Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
- PATIENT EXCLUSION CRITERIA TO PROCEED TO HCT:
- Patients with uncontrolled fungal, bacterial, viral, or parasitic infections are excluded. Patients with history of fungal disease during chemotherapy may proceed if they have a significant response to antifungal therapy with no or minimal evidence of disease remaining by computed tomography (CT) evaluation
- Patients with active central nervous system (CNS) leukemia or any other active site of extramedullary disease at the time of initiation of the conditioning regimen are not permitted.
- Note: Those with prior history of CNS or extramedullary disease, but with no active disease at the time of pre-transplant workup, are eligible
- Pregnant or breastfeeding females are ineligible as many of the medications used in this protocol could be harmful to unborn children and infants
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (63 Sites)
Showing the first 25 of 63 sites.
- Children's Hospital of Alabama — Birmingham, Alabama
- Phoenix Childrens Hospital — Phoenix, Arizona
- Arkansas Children's Hospital — Little Rock, Arkansas
- City of Hope Comprehensive Cancer Center — Duarte, California
- Loma Linda University Medical Center — Loma Linda, California
- Children's Hospital Los Angeles — Los Angeles, California
- UCSF Benioff Children's Hospital Oakland — Oakland, California
- Lucile Packard Children's Hospital Stanford University — Palo Alto, California
- UCSF Medical Center-Mission Bay — San Francisco, California
- Children's Hospital Colorado — Aurora, Colorado
- Yale University — New Haven, Connecticut
- Alfred I duPont Hospital for Children — Wilmington, Delaware
- Children's National Medical Center — Washington D.C., District of Columbia
- UF Health Cancer Institute - Gainesville — Gainesville, Florida
- Nemours Children's Clinic-Jacksonville — Jacksonville, Florida
- University of Miami Miller School of Medicine-Sylvester Cancer Center — Miami, Florida
- Nicklaus Children's Hospital — Miami, Florida
- AdventHealth Orlando — Orlando, Florida
- Johns Hopkins All Children's Hospital — St. Petersburg, Florida
- Lurie Children's Hospital-Chicago — Chicago, Illinois
- University of Chicago Comprehensive Cancer Center — Chicago, Illinois
- Riley Hospital for Children — Indianapolis, Indiana
- University of Iowa/Holden Comprehensive Cancer Center — Iowa City, Iowa
- Norton Children's Hospital — Louisville, Kentucky
- Children's Hospital New Orleans — New Orleans, Louisiana
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
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💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.