NCT07566377

Brief Summary

The purpose of this study is to find out whether Cord Blood Transplantation/CBT as the first or second transplant is an effective treatment for children and young adults with blood cancer.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
71

participants targeted

Target at P50-P75 for phase_2

Timeline
49mo left

Started Apr 2026

Typical duration for phase_2

Geographic Reach
1 country

7 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress1%
Apr 2026Apr 2030

Study Start

First participant enrolled

April 28, 2026

Completed
1 day until next milestone

First Submitted

Initial submission to the registry

April 29, 2026

Completed
6 days until next milestone

First Posted

Study publicly available on registry

May 5, 2026

Completed
4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 28, 2030

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 28, 2030

Last Updated

May 5, 2026

Status Verified

April 1, 2026

Enrollment Period

4 years

First QC Date

April 29, 2026

Last Update Submit

April 29, 2026

Conditions

Keywords

Cord blood transplantsGraft-versus-host DiseaseLymphomaLeukemiaHodgkin LymphomaNon-hodgkin LymphomaMyelodysplastic SyndromesAcute Lymphoblastic LeukemiaAcute Myelogenous LeukemiaMemorial Sloan Kettering Cancer Center26-168

Outcome Measures

Primary Outcomes (1)

  • Disease-free Survival (DFS)

    Disease-free Survival (DFS) at 1 year after CBT

    1 year

Study Arms (6)

Cohort 1: Patients with High-Risk Disease + Regimen A

EXPERIMENTAL

Participants in complete remission (CR; bone marrow blasts \<5% by morphology) with no prior allogeneic transplant, who require allogeneic transplantation and do not have human leukocyte antigen (HLA)-matched related or unrelated donors readily available within 4 weeks. For participants with AML/MDS, MRD (Measurable/Minimal Residual Disease) positive status at the time of transplant is accepted (evaluated by multiparameter flow cytometry); participants with ALL need to be in MRD negative status (evaluated by multiparameter flow cytometry).

Biological: Cord Blood UnitsRadiation: Total Body IrradiationDrug: CyclophosphamideDrug: FludarabineDrug: ClofarabineDrug: BusulfanDrug: ThiotepaDrug: TacrolimusDrug: Mycophenolate MofetilDrug: Cyclosporine

Cohort 1: Patients with High-Risk Disease + Regimen B

EXPERIMENTAL

Participants in complete remission (CR; bone marrow blasts \<5% by morphology) with no prior allogeneic transplant, who require allogeneic transplantation and do not have human leukocyte antigen (HLA)-matched related or unrelated donors readily available within 4 weeks. For participants with AML/MDS, MRD (Measurable/Minimal Residual Disease) positive status at the time of transplant is accepted (evaluated by multiparameter flow cytometry); participants with ALL need to be in MRD negative status (evaluated by multiparameter flow cytometry).

Biological: Cord Blood UnitsRadiation: Total Body IrradiationDrug: CyclophosphamideDrug: FludarabineDrug: ClofarabineDrug: BusulfanDrug: ThiotepaDrug: TacrolimusDrug: Mycophenolate MofetilDrug: Cyclosporine

Cohort 1: Patients with High-Risk Disease + Regimen C

EXPERIMENTAL

Participants in complete remission (CR; bone marrow blasts \<5% by morphology) with no prior allogeneic transplant, who require allogeneic transplantation and do not have human leukocyte antigen (HLA)-matched related or unrelated donors readily available within 4 weeks. For participants with AML/MDS, MRD (Measurable/Minimal Residual Disease) positive status at the time of transplant is accepted (evaluated by multiparameter flow cytometry); participants with ALL need to be in MRD negative status (evaluated by multiparameter flow cytometry).

Biological: Cord Blood UnitsRadiation: Total Body IrradiationDrug: CyclophosphamideDrug: FludarabineDrug: ClofarabineDrug: BusulfanDrug: ThiotepaDrug: TacrolimusDrug: Mycophenolate MofetilDrug: Cyclosporine

Cohort 2: Patients with Very High-Risk Disease + Regimen A

EXPERIMENTAL

1. Participants in CR (bone marrow blasts \<5% by morphology) who had prior allogeneic transplant and disease recurrence. 1. Participants with AML/MDS: MRD positive status at the time of transplant is accepted (evaluated by multiparameter flow cytometry) 2. Participants with ALL: MRD positive status at the time of transplant is accepted (evaluated by multiparameter flow cytometry). 3. The second transplant will take place at least 4 months after the first. 2. Participants with relapsed/refractory disease at first or second allogeneic transplant, with up to 30% bone marrow blasts by multiparameter flow cytometry or morphology.

Biological: Cord Blood UnitsRadiation: Total Body IrradiationDrug: CyclophosphamideDrug: FludarabineDrug: ClofarabineDrug: BusulfanDrug: ThiotepaDrug: TacrolimusDrug: Mycophenolate MofetilDrug: Cyclosporine

Cohort 2: Patients with Very High-Risk Disease + Regimen B

EXPERIMENTAL

1. Participants in CR (bone marrow blasts \<5% by morphology) who had prior allogeneic transplant and disease recurrence. 1. Participants with AML/MDS: MRD positive status at the time of transplant is accepted (evaluated by multiparameter flow cytometry) 2. Participants with ALL: MRD positive status at the time of transplant is accepted (evaluated by multiparameter flow cytometry). 3. The second transplant will take place at least 4 months after the first. 2. Participants with relapsed/refractory disease at first or second allogeneic transplant, with up to 30% bone marrow blasts by multiparameter flow cytometry or morphology.

Biological: Cord Blood UnitsRadiation: Total Body IrradiationDrug: CyclophosphamideDrug: FludarabineDrug: ClofarabineDrug: BusulfanDrug: ThiotepaDrug: TacrolimusDrug: Mycophenolate MofetilDrug: Cyclosporine

Cohort 2: Patients with Very High-Risk Disease + Regimen C

EXPERIMENTAL

1. Participants in CR (bone marrow blasts \<5% by morphology) who had prior allogeneic transplant and disease recurrence. 1. Participants with AML/MDS: MRD positive status at the time of transplant is accepted (evaluated by multiparameter flow cytometry) 2. Participants with ALL: MRD positive status at the time of transplant is accepted (evaluated by multiparameter flow cytometry). 3. The second transplant will take place at least 4 months after the first. 2. Participants with relapsed/refractory disease at first or second allogeneic transplant, with up to 30% bone marrow blasts by multiparameter flow cytometry or morphology.

Biological: Cord Blood UnitsRadiation: Total Body IrradiationDrug: CyclophosphamideDrug: FludarabineDrug: ClofarabineDrug: BusulfanDrug: ThiotepaDrug: TacrolimusDrug: Mycophenolate MofetilDrug: Cyclosporine

Interventions

Cord Blood \[(HPC(CB)\] products are minimally manipulated unrelated allogeneic cord blood units that have been collected, processed and stored in public Cord Blood banks

Cohort 1: Patients with High-Risk Disease + Regimen ACohort 1: Patients with High-Risk Disease + Regimen BCohort 1: Patients with High-Risk Disease + Regimen CCohort 2: Patients with Very High-Risk Disease + Regimen ACohort 2: Patients with Very High-Risk Disease + Regimen BCohort 2: Patients with Very High-Risk Disease + Regimen C

Hyper-fractionated TBI is administered by a linear accelerator at a dose rate of \<20 cGy/minute. Treatment planning begins with simulation.

Also known as: TBI
Cohort 1: Patients with High-Risk Disease + Regimen ACohort 1: Patients with High-Risk Disease + Regimen BCohort 1: Patients with High-Risk Disease + Regimen CCohort 2: Patients with Very High-Risk Disease + Regimen ACohort 2: Patients with Very High-Risk Disease + Regimen BCohort 2: Patients with Very High-Risk Disease + Regimen C

Cyclophosphamide is an alkylating agent that prevents cell division by cross-linking DNA strands and decreasing DNA synthesis.

Also known as: Cytoxan, Neosar
Cohort 1: Patients with High-Risk Disease + Regimen ACohort 1: Patients with High-Risk Disease + Regimen BCohort 1: Patients with High-Risk Disease + Regimen CCohort 2: Patients with Very High-Risk Disease + Regimen ACohort 2: Patients with Very High-Risk Disease + Regimen BCohort 2: Patients with Very High-Risk Disease + Regimen C

Fludarabine phosphate is rapidly dephosphorylated to 2- fluoro-ara- A and then phosphorylated intracellularly by deoxycytidine kinase to the active triphosphate, 2- fluoro-ara-ATP

Also known as: Fludara
Cohort 1: Patients with High-Risk Disease + Regimen ACohort 1: Patients with High-Risk Disease + Regimen BCohort 1: Patients with High-Risk Disease + Regimen CCohort 2: Patients with Very High-Risk Disease + Regimen ACohort 2: Patients with Very High-Risk Disease + Regimen BCohort 2: Patients with Very High-Risk Disease + Regimen C

Clofarabine, a purine (deoxyadenosine) nucleoside analog, is metabolized to clofarabine 5'-triphosphate.

Also known as: Clolar
Cohort 1: Patients with High-Risk Disease + Regimen ACohort 1: Patients with High-Risk Disease + Regimen BCohort 1: Patients with High-Risk Disease + Regimen CCohort 2: Patients with Very High-Risk Disease + Regimen ACohort 2: Patients with Very High-Risk Disease + Regimen BCohort 2: Patients with Very High-Risk Disease + Regimen C

Busulfan is a bifunctional alkylating agent known chemically as 1,4- butanediol, dimethanesulfonate.

Also known as: Busulfex
Cohort 1: Patients with High-Risk Disease + Regimen ACohort 1: Patients with High-Risk Disease + Regimen BCohort 1: Patients with High-Risk Disease + Regimen CCohort 2: Patients with Very High-Risk Disease + Regimen ACohort 2: Patients with Very High-Risk Disease + Regimen BCohort 2: Patients with Very High-Risk Disease + Regimen C

Thiotepa is an alkylating agent which produces cross-linking of DNA strands leading to inhibition of DNA, RNA, and protein synthesis; thiotepa is cell-cycle independent.

Also known as: Thioplex
Cohort 1: Patients with High-Risk Disease + Regimen ACohort 1: Patients with High-Risk Disease + Regimen BCohort 1: Patients with High-Risk Disease + Regimen CCohort 2: Patients with Very High-Risk Disease + Regimen ACohort 2: Patients with Very High-Risk Disease + Regimen BCohort 2: Patients with Very High-Risk Disease + Regimen C

Tacrolimus inhibits T-lymphocyte activation

Also known as: Prograf
Cohort 1: Patients with High-Risk Disease + Regimen ACohort 1: Patients with High-Risk Disease + Regimen BCohort 1: Patients with High-Risk Disease + Regimen CCohort 2: Patients with Very High-Risk Disease + Regimen ACohort 2: Patients with Very High-Risk Disease + Regimen BCohort 2: Patients with Very High-Risk Disease + Regimen C

Mycophenolate exhibits a cytostatic effect on T and B lymphocytes.

Also known as: CellCept
Cohort 1: Patients with High-Risk Disease + Regimen ACohort 1: Patients with High-Risk Disease + Regimen BCohort 1: Patients with High-Risk Disease + Regimen CCohort 2: Patients with Very High-Risk Disease + Regimen ACohort 2: Patients with Very High-Risk Disease + Regimen BCohort 2: Patients with Very High-Risk Disease + Regimen C

Cyclosporine is a calcineurin inhibitor that inhibits production and release of interleukin II and inhibits interleukin II-induced activation of resting T-lymphocytes.

Cohort 1: Patients with High-Risk Disease + Regimen ACohort 1: Patients with High-Risk Disease + Regimen BCohort 1: Patients with High-Risk Disease + Regimen CCohort 2: Patients with Very High-Risk Disease + Regimen ACohort 2: Patients with Very High-Risk Disease + Regimen BCohort 2: Patients with Very High-Risk Disease + Regimen C

Eligibility Criteria

AgeUp to 26 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • A patient cannot be considered eligible for this study unless ALL of the following conditions are met.
  • ° Disease type
  • Cohort 1, High Risk Disease: Patients with age ≤ 26 years at the time of informed consent with no available and suitably matched related or unrelated donor within 4 weeks, with one of the following diagnoses:
  • I. Acute myelogenous leukemia (AML):
  • Complete first remission (CR1) with blast count \< 5% by bone marrow morphology at high risk for relapse such as any of the following:
  • Known prior diagnosis of myelodysplasia (MDS)
  • High risk cytogenetics (e.g., those associated with MDS, abnormalities of 5, 7, 8, complex karyotype) and/or high-risk molecular abnormalities (e.g., TP53)
  • Requirement for 2 or more inductions to achieve CR1
  • Therapy-related AML (t-AML) or therapy-related myeloid neoplasm (t-MN) (including after therapy for other malignancy, and/or gene therapy or cell therapy)
  • Presence of Minimal/Measurable Residual Disease (MRD+) by cytogenetics, flow cytometry or molecular methods (at End of Induction or End of Consolidation)
  • Other high-risk features not defined above.
  • Complete second remission (CR2) or subsequent remission, with blast count \< 5% by bone marrow morphology
  • Presence of MRD by multiparameter flow cytometry at pre-transplant evaluation is acceptable.
  • II. Acute lymphoblastic leukemia (ALL):
  • Complete first remission (CR1) with MRD negative status by multicolor flow cytometry, at high risk for relapse such as any of the following:
  • +42 more criteria

You may not qualify if:

  • ° Inadequate performance status/ organ function.
  • ° Active CNS leukemic involvement.
  • Chloroma \>2 cm.
  • Active and uncontrolled infection (bacterial/fungal/viral) at time of transplant.
  • HIV infection.
  • Seropositivity for HTLV-1.
  • Pregnancy or breast feeding.
  • Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, long-term follow-up, and research tests.
  • Any abnormal condition or lab result that is considered by the PI capable or altering patient's condition or study outcome.
  • Cohort 2 Very High-Risk Disease (additional to above):
  • ° Allogeneic HCT in the preceding 4 months.
  • Note (1): Prior checkpoint inhibitors/blockade in the last 12 months: eligibility to be discussed with study PI.
  • Note (2): For patients with known HBV and/or HCV infection :
  • For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated.
  • Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Memorial Sloan Kettering Basking Ridge (Limited Protocol Activities)

Basking Ridge, New Jersey, 07920, United States

RECRUITING

Memorial Sloan Kettering Monmouth (Limited Protocol Activities)

Middletown, New Jersey, 07748, United States

RECRUITING

Memorial Sloan Kettering Bergen (Limited Protocol Activities)

Montvale, New Jersey, 07645, United States

RECRUITING

Memorial Sloan Kettering Suffolk - Commack (Limited Protocol Activities)

Commack, New York, 11725, United States

RECRUITING

Memorial Sloan Kettering Westchester (Limited Protocol Activities)

Harrison, New York, 10604, United States

RECRUITING

Memorial Sloan Kettering Cancer Center (All Protocol Activities)

New York, New York, 10065, United States

RECRUITING

Memorial Sloan Kettering Nassau (Limited Protocol Activities)

Rockville Centre, New York, 11553, United States

RECRUITING

Related Links

MeSH Terms

Conditions

Leukemia, Myeloid, AcutePrecursor Cell Lymphoblastic Leukemia-LymphomaMyelodysplastic SyndromesLymphoma, Non-HodgkinHodgkin DiseaseLeukemiaLymphomaGraft vs Host Disease

Interventions

Whole-Body IrradiationCyclophosphamidefludarabinefludarabine phosphateClofarabineBusulfanThiotepaTacrolimusMycophenolic AcidCyclosporine

Condition Hierarchy (Ancestors)

Leukemia, MyeloidNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLeukemia, LymphoidLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesBone Marrow Diseases

Intervention Hierarchy (Ancestors)

RadiotherapyTherapeuticsInvestigative TechniquesPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsAdenine NucleotidesPurine NucleotidesPurinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsArabinonucleosidesNucleosidesNucleic Acids, Nucleotides, and NucleosidesNucleotidesRibonucleotidesButylene GlycolsGlycolsAlcoholsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicSulfonic AcidsSulfur AcidsSulfur CompoundsTriethylenephosphoramideAziridinesAzirinesHeterocyclic Compounds, 1-RingMacrolidesLactonesCaproatesAcids, AcyclicCarboxylic AcidsFatty AcidsLipidsCyclosporinsPeptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesAmino Acids, Peptides, and Proteins

Study Officials

  • Andromachi Scaradavou, MD

    Memorial Sloan Kettering Cancer Center

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Andromachi Scaradavou, MD

CONTACT

Jaap Jan Boelens, MD, PhD

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 29, 2026

First Posted

May 5, 2026

Study Start

April 28, 2026

Primary Completion (Estimated)

April 28, 2030

Study Completion (Estimated)

April 28, 2030

Last Updated

May 5, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made following one year after publication and for up to 36 months later. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

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