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Which stem cell donor is best for kids with leukemia? major trial aims to find out

NCT ID NCT05457556

Disease control Sponsor: Children's Oncology Group Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 31, 2026 · Updated 4 times

Summary

This phase 3 trial compares two types of stem cell transplants for children, adolescents, and young adults with acute leukemia or myelodysplastic syndrome (MDS). One uses a half-matched family donor (haploidentical), the other a fully matched unrelated donor. The goal is to see which approach leads to better survival and fewer severe side effects like graft-versus-host disease. About 435 participants will be randomly assigned to one of the two transplant types.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
stem cell transplant (from a haploidentical related donor or matched unrelated donor)
What this could lead to
If successful, this trial could show that using a half-matched family donor is as good as or better than a fully matched unrelated donor for stem cell transplants in children and young adults with leukemia or MDS.
What could go wrong
This is a large phase 3 trial, but it compares two existing transplant approaches—neither is a new treatment. The results may show no clear winner, and transplants carry serious risks like graft-versus-host disease and infection.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

66 people

The number who actually took part.

Started

Mar 2023

Expected to finish

Jul 2027

An estimate. End dates often move.

Lead sponsor

A research network

The lead sponsor is a research network or cooperative group.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 months to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * PATIENT INCLUSION CRITERIA FOR ENROLLMENT: * 6 months to \< 22 years at enrollment * Diagnosed with ALL, AML, or MDS or mixed phenotype acute leukemia (MPAL) for which an allogeneic hematopoietic stem cell transplant is indicated. Complete Remission (CR) status will not be confirmed at the time of enrollment. CR as defined in these sections is required to proceed with the actual HCT treatment plan * Has not received a prior allogeneic hematopoietic stem cell transplant * Does not have a suitable human leukocyte antigen (HLA)-matched sibling donor available for stem cell donation * Has an eligible haploidentical related family donor based on at least intermediate resolution HLA typing * Patients who also have an eligible 8/8 MUD adult donor based on confirmatory high resolution HLA typing are eligible for randomization to Arm A or Arm B. * Patients who do not have an eligible MUD donor are eligible for enrollment to Arm C * All patients and/or their parents or legal guardians must sign a written informed consent * All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met * Co-Enrollment on other trials * Patients will not be excluded from enrollment on this study if already enrolled on other protocols for treatment of high risk and/or relapsed ALL, AML, MPAL and MDS. This is including, but not limited to, COG AAML1831, COG AALL1821, the EndRAD Trial, as well as local institutional trials. We will collect information on all co-enrollments * Patients will not be excluded from enrollment on this study if receiving immunotherapy prior to transplant as a way to achieve remission and bridge to transplant. This includes chimeric antigen receptor (CAR) T cell therapy and other immunotherapies * PATIENT INCLUSION CRITERIA TO PROCEED TO HCT: * Karnofsky Index or Lansky Play-Performance Scale \>= 60 on pre-transplant evaluation. Karnofsky scores must be used for patients \>= 16 years of age and Lansky scores for patients =\< 16 years of age (within 4 weeks of starting therapy) * A serum creatinine based on age/gender as follows: 6 months to \< 1 year: 0.5 mg/dL (Male); 0.5 mg/dL (Female) 1. to \< 2 years: 0.6 mg/dL (Male); 0.6 mg/dL (Female) 2. to \< 6 years: 0.8 mg/dL (Male); 0.8 mg/dL (Female) 6 to \< 10 years: 1 mg/dL (Male); 1 mg/dL (Female) 10 to \< 13 years: 1.2 mg/dL (Male); 1.2 mg/dL (Female) 13 to \< 16 years: 1.5 mg/dL (Male); 1.4 mg/dL (Female) \>= 16 years: 1.7 mg/dL (Male); 1.4 mg/dL (Female) * OR * A 24 hour urine Creatinine clearance \>= 60 mL/min/1.73 m\^2 * OR * A glomerular filtration rate (GFR) \>= 60 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard) * Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility * Serum glutamic-oxaloacetic transaminase (SGOT) aspartate aminotransferase \[AST\] or serum glutamate pyruvate transaminase (SGPT) aminotransferase \[ALT\] \< 5 x upper limit of normal (ULN) for age * Total bilirubin \< 2.5 mg/dL, unless attributable to Gilbert's Syndrome * Shortening fraction of \>= 27% by echocardiogram or radionuclide scan (MUGA) * OR * Ejection fraction of \>= 50% by echocardiogram or radionuclide scan (MUGA), choice of test according to local standard of care * Forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and corrected carbon monoxide diffusing capability (DLCO) must all be \>= 50% of predicted by pulmonary function tests (PFTs). * For children who are unable to perform for PFTs (e.g., due to age or developmental delay), the criteria are: no evidence of dyspnea at rest, oxygen (O2) saturation (Sat) \> 92% on room air by pulse oximetry, not on supplemental O2 at rest, and not on supplemental O2 at rest * MPAL in first complete remission (CR1) for whom transplant is indicated. Examples include those patients who are poorly responsive to ALL therapy (end of induction failure( IF-MPAL) to ALL induction (see IF-MPAL note below), end of induction MRD ≥ 5% or end-of-consolidation MRD \> 0.01%), as well as patients treated with AML therapy * IF-MPAL: additional criterion for Induction failure for MPAL ONLY as per ALL1732: * An increasing number of circulating leukemia cells on 3 or more consecutive CBCs obtained at daily or longer intervals following day 8 of Induction therapy and prior to day 29 with confirmation by flow cytometry OR development of new sites of extramedullary disease, or other laboratory or clinical evidence of refractory disease or progression prior to the end of Induction evaluation (note that residual testicular disease at the end of Induction is an exception) * MPAL in \> second complete remission (CR2) * ALL high-risk in CR1 for whom transplant is indicated. Examples include: induction failure, treatment failure as per minimal residual disease by flow cytometry \> 0.01% after consolidation and not eligible for AALL1721 or AALL1721 not available/unwilling to enroll, hypodiploidy (\< 44 chromosomes) with MRD+ \> 0.01% after induction, persistent or recurrent cytogenetic or molecular evidence of disease during therapy requiring additional therapy after induction to achieve remission (e.g. persistent molecular BCR-ABL positivity), T cell ALL with persistent MRD \> 0.01% after consolidation. * ALL in CR2 for whom transplant is indicated. Examples include: B-cell: early (=\< 36 months from initiation of therapy) bone marrow (BM) relapse, late BM relapse (\>= 36 months) with MRD \>= 0.1% by flow cytometry after first re-induction therapy; T or B-cell: early (\< 18 months) isolated extramedullary (IEM), late (\>= 18 months) IEM, end-Block 1 MRD \>= 0.1%; T-cell or Philadelphia chromosome positive (Ph+): BM relapse at any time * ALL in \>= third complete remission (CR3) * Patients treated with chimeric antigen receptor T-cells (CART) cells for whom transplant is indicated. Examples include: transplant for consolidation of CART, loss of CART persistence and/or B cell aplasia \< 6 months from infusion or have other evidence (e.g., MRD+) that transplant is indicated to prevent relapse * AML in CR1 for whom transplant is indicated. Examples include those deemed high risk for relapse as described in AAML1831: * FLT3/ITD+ with allelic ratio \> 0.1 without bZIP CEBPA, NPM1 * FLT3/ITD+ with allelic ratio \> 0.1 with concurrent bZIP CEBPA or NPM1 and with evidence of residual AML (MRD \>= 0.05%) at end of Induction * Presence of RAM phenotype or unfavorable prognostic markers (other than FLT3/ITD) per cytogenetics, fluorescence in situ hybridization (FISH), next generation sequencing (NGS) results, regardless of favorable genetic markers, MRD status or FLT3/ITD mutation status * AML without favorable or unfavorable cytogenetic or molecular features but with evidence of residual AML (MRD \>= 0.05%) at end of Induction * Presence of a non-ITD FLT3 activating mutation and positive MRD (\>= 0.05%) at end of Induction 1 regardless of presence of favorable genetic markers. * AML in \>= CR2 * MDS with \< 5% blasts by morphology and flow cytometry (if available) on the pre-transplant bone marrow evaluation * Complete remission (CR) is defined as \< 5% blasts by morphology and flow cytometry (if available) on the pre-transplant bone marrow evaluation with minimum sustained absolute neutrophil count (ANC) of 300 cells/microliter for 1 week or ANC \> 500 cells/microliter. We will be collecting data from all approaches to MRD evaluation performed including NGS and polymerase chain reaction (PCR). It is strongly recommended that MPAL be evaluated using multidimensional flow cytometry and/or (KMT2Ar) qt PCR. It is strongly recommended that MPAL be evaluated using multidimensional flow cytometry and/or (KMT2Ar) qt PCR * DONOR ELIGIBILITY CRITERIA: * Matched Unrelated Donors: Unrelated donor candidates must be matched at high resolution at a minimum of 8/8 alleles (HLA-A, -B, -C, -DRB1). One-antigen HLA mismatches are not permitted. HLA matching of additional alleles is recommended according to National Marrow Donor Program (NMDP) guidelines, but will be at the discretion of local centers * Haploidentical Matched Family Members: * Minimum match level full haploidentical (at least 5/10; HLA-A, -B, -C, -DRB1, -DQB1 alleles). The following issues (in no particular order) should be considered in choosing a haploidentical donor: * Absent or low patient donor-specific antibodies (DSA) * Mean fluorescence intensity (MFI) of any anti-donor HLA antibody by solid phase immunoassay should be \< 2000. Donors with higher levels are not eligible. * If a screening assay against pooled HLA antigens is used, positive results must be followed with specificity testing using a single antigen assay. The MFI must be \< 2000 unless the laboratory has validated higher threshold values for reactivity for HLA antigens (such as HLA-C, -DQ, and -DP), that may be enhanced in concentration on the single antigen assays. Donor anti- recipient antibodies are of unknown clinical significance and do not need to be sent or reported. * Consult with Study Chair for the clinical significance of any recipient anti-donor HLA antibody. * If centers are unable to perform this type of testing, please contact the Study Chair to make arrangements for testing. * If killer immunoglobulin testing (KIR) is performed: KIR status by mismatch, KIR-B, or KIR content criteria can be used according to institutional guidelines. * ABO compatibility (in order of priority): * Compatible or minor ABO incompatibility * Major ABO incompatibility * CMV serostatus: * For a CMV seronegative recipient: the priority is to use a CMV seronegative donor when feasible * For a CMV seropositive recipient: the priority is to use a CMV seropositive donor when feasible * Age: younger donors including siblings/half-siblings, and second degree relatives (aunts, uncles, cousins) are recommended, even if \< 18 years * Size and vascular access appropriate by center standard for peripheral blood stem cell (PBSC) collection if needed * Haploidentical matched family members: screened by center health screens and found to be eligible * Unrelated donors: meet eligibility criteria as defined by the NMDP or other unrelated donor registries. If the donor does not meet the registry eligibility criteria but an acceptable eligibility waiver is completed and signed per registry guidelines, the donor will be considered eligible for this study * Human immunodeficiency virus (HIV) negative * Not pregnant * MUD donors and post-transplant cyclophosphamide haplo donors should be asked to provide BM. If donors refuse and other donors are not available, PBSC is allowed. TCR-alpha beta/CD19 depleted haplo donors must agree to donate PBSC * Must give informed consent: * Haploidentical matched family members: Institution standard of care donor consent and Protocol-specific Donor Consent for Optional Studies * Unrelated donors: standard NMDP Unrelated Donor Consent 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

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Conditions

The condition(s) this trial relates to.

acute biphenotypic leukemia acute lymphoblastic leukemia acute myeloid leukemia mixed phenotype acute leukemia myelodysplastic syndrome Myelodysplastic Syndromes Precursor Cell Lymphoblastic Leukemia-Lymphoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Acute lymphoblastic leukemia Acute myeloid leukemia Mixed phenotype acute leukemia Myelodysplastic syndrome

Contacts and locations

Locations

  • AdventHealth Orlando

    Orlando, Florida, 32803, United States

  • Alberta Children's Hospital

    Calgary, Alberta, T3B 6A8, Canada

  • Alfred I duPont Hospital for Children

    Wilmington, Delaware, 19803, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202-3591, United States

  • Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

    Houston, Texas, 77030, United States

  • British Columbia Children's Hospital

    Vancouver, British Columbia, V6H 3V4, Canada

  • C S Mott Children's Hospital

    Ann Arbor, Michigan, 48109, United States

  • CancerCare Manitoba

    Winnipeg, Manitoba, R3E 0V9, Canada

  • Carolinas Medical Center/Levine Cancer Institute

    Charlotte, North Carolina, 28203, United States

  • Centre Hospitalier Universitaire Sainte-Justine

    Montreal, Quebec, H3T 1C5, Canada

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital New Orleans

    New Orleans, Louisiana, 70118, United States

  • Children's Hospital of Alabama

    Birmingham, Alabama, 35233, United States

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201, United States

  • Children's Mercy Hospitals and Clinics

    Kansas City, Missouri, 64108, United States

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • City of Hope Comprehensive Cancer Center

    Duarte, California, 91010, United States

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104, United States

  • Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital

    Grand Rapids, Michigan, 49503, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701, United States

  • Johns Hopkins University/Sidney Kimmel Cancer Center

    Baltimore, Maryland, 21287, United States

  • Laura and Isaac Perlmutter Cancer Center at NYU Langone

    New York, New York, 10016, United States

  • Loma Linda University Medical Center

    Loma Linda, California, 92354, United States

  • Lucile Packard Children's Hospital Stanford University

    Palo Alto, California, 94304, United States

  • Lurie Children's Hospital-Chicago

    Chicago, Illinois, 60611, United States

  • Mayo Clinic in Rochester

    Rochester, Minnesota, 55905, United States

  • Medical City Dallas Hospital

    Dallas, Texas, 75230, United States

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Methodist Children's Hospital of South Texas

    San Antonio, Texas, 78229, United States

  • Montefiore Medical Center - Moses Campus

    The Bronx, New York, 10467, United States

  • NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

    New York, New York, 10032, United States

  • Nemours Children's Clinic-Jacksonville

    Jacksonville, Florida, 32207, United States

  • New York Medical College

    Valhalla, New York, 10595, United States

  • Nicklaus Children's Hospital

    Miami, Florida, 33155, United States

  • Norton Children's Hospital

    Louisville, Kentucky, 40202, United States

  • Penn State Children's Hospital

    Hershey, Pennsylvania, 17033, United States

  • Phoenix Childrens Hospital

    Phoenix, Arizona, 85016, United States

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113, United States

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202, United States

  • Roswell Park Cancer Institute

    Buffalo, New York, 14263, United States

  • The Children's Hospital at TriStar Centennial

    Nashville, Tennessee, 37203, United States

  • The Children's Hospital at Westmead

    Westmead, New South Wales, 2145, Australia

  • The Steven and Alexandra Cohen Children's Medical Center of New York

    New Hyde Park, New York, 11040, United States

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609, United States

  • UCSF Medical Center-Mission Bay

    San Francisco, California, 94158, United States

  • UF Health Cancer Institute - Gainesville

    Gainesville, Florida, 32610, United States

  • UT Southwestern/Simmons Cancer Center-Dallas

    Dallas, Texas, 75390, United States

  • University of Chicago Comprehensive Cancer Center

    Chicago, Illinois, 60637, United States

  • University of Iowa/Holden Comprehensive Cancer Center

    Iowa City, Iowa, 52242, United States

  • University of Miami Miller School of Medicine-Sylvester Cancer Center

    Miami, Florida, 33136, United States

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216, United States

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104, United States

  • University of Rochester

    Rochester, New York, 14642, United States

  • University of Wisconsin Carbone Cancer Center - University Hospital

    Madison, Wisconsin, 53792, United States

  • VCU Massey Comprehensive Cancer Center

    Richmond, Virginia, 23298, United States

  • Vanderbilt University/Ingram Cancer Center

    Nashville, Tennessee, 37232, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

  • Yale University

    New Haven, Connecticut, 06520, United States

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