Cord Blood Transplantation in Children and Young Adults With Blood Cancer

May 11, 2026 updated by: Memorial Sloan Kettering Cancer Center

Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

71

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • New York
      • New York, New York, United States, 10065
        • Recruiting
        • Memorial Sloan Kettering Cancer Center (All Protocol Activities)
        • Contact:
          • Andromachi Scaradavou, MD
          • Phone Number: 1-833-MSK-KIDS

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

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:

    • Presence of any high risk cytogenetic abnormalities such as t(9;22), t(1;19), t(4;11) or other, KMT2A (11q23) or other high risk molecular abnormality
    • Failure to achieve complete remission (CR) after four weeks of induction therapy (transplant to follow antibody therapy and/or CAR T cells)
    • Persistence or recurrence of MRD on therapy (Transplant to follow antibody therapy and/or CAR T cells)
    • T-ALL in CR even with presence of MRD
    • Other high-risk features not defined above

  • Complete second remission (CR2) or subsequent remission with MRD negative status by multiparameter flow cytometry.

    • Relapse in less than 36 months from CR1
    • Relapse for T-ALL
  • Patients after antibody therapy (e.g., blinatumomab, inotuzumab, other) and/or CAR-T cell therapy that resulted in MRD negative status by multiparameter flow cytometry.

III. Other acute leukemias:

  • Leukemias of ambiguous lineage or of other types with < 5% blasts by bone marrow morphology.
  • Patients with persistent/relapsed disease with cytogenetic, flow cytometric or molecular aberrations in < 5% of cells.
  • Chronic myelogenous leukemia: Patients with history of blast crisis or accelerated phase.
  • Any leukemia that developed after gene therapy or cell therapy

IV. Myelodysplastic Syndrome (MDS):

  • Any IPSS risk category with life-threatening cytopenia(s).
  • Any IPSS risk category with high risk cytogenetic/molecular findings (5, 7, 8, complex karyotype, or TP53)

V. Non-Hodgkin lymphoma (NHL) or Hodgkin lymphoma (HL) at high risk of relapse or progression if not in remission:

  • Patients with aggressive histology (such as, but not limited to, diffuse large B-cell NHL, mantle cell NHL, and T-cell NHL) in CR.
  • Patients with indolent B cell NHL (such as, but not limited to, follicular, small cell or marginal zone NHL) will have 2nd or subsequent progression with stable disease/ CR/ PR with no single lesion equal to or more than 5 cm.
  • Patients with HL without progression of disease (POD) after salvage chemotherapy with no single lesion ≥5 cm.

Cohort 2: Very High-Risk disease:

  1. Patients in CR (bone marrow blasts <5% by morphology) who had prior allogeneic transplant and disease recurrence. The second transplant will take place at least 4 months after the first.

    • Acute myelogenous leukemia (AML) or Myelodysplastic Syndrome (MDS): Relapse after previous transplant, in CR after induction therapy. MRD positive status by multi-parameter flow cytometry is accepted.
    • Acute lymphoblastic leukemia (ALL): Relapse after previous transplant, in CR after induction therapy and/or antibody therapy/CAR T cells. MRD positive status after targeted therapy, as evaluated by multi-parameter flow cytometry is accepted.
    • Other: patients with leukemia or lymphoma, who, in the opinion of their physician, are not likely to have reduction in disease burden with further chemotherapy.

  2. Patients with relapsed/refractory disease at either first or second allogeneic transplant, with up to 30% bone marrow blasts by multiparameter flow cytometry or morphology. ° Relapse after previous transplant with < 30% blasts by bone marrow morphology, or with cytogenetic, flow cytometric, or molecular abnormalities in < 30% of bone marrow cells, after induction therapy.

    ° Primary refractory or relapsed AML with < 30% blasts by bone marrow morphology or with cytogenetic, flow cytometric, or molecular abnormalities in < 30% of bone marrow cells.

    ° Age 0-26 years at the time of informed consent

    ° Performance: Karnofsky (≥16 years) or Lansky score (<16 years) of ≥70% (see Appendix A).

    ° Not Pregnant and Not Nursing

    ° Required Organ Function

    • Bilirubin ≤ 1.5 mg/dL (unless benign congenital hyperbilirubinemia).
    • ALT ≤ 3 x upper limit of normal.

    • Pulmonary function (FVC, FEV1 and DLCO corrected for hemoglobin) ≥ 50% predicted.

      • In young children unable to perform pulmonary function testing: pulse oximetry >92% in room air, and a normal CT of the chest (if CT is not normal, the child needs to be evaluated and cleared by pediatric pulmonary physician).
    • Left ventricular ejection fraction > 50%.
    • Age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index (aaHCT-CI) ≤ 7.
    • Female patients of childbearing potential must have a negative serum pregnancy test within 7 days of enrolment and must be willing to use an effective contraceptive method while enrolled in the study.
    • Renal: Serum creatinine (SCr) ≤ 1.5 x normal for age. If SCr is outside the normal range, then CrCl > 50 mL/min (calculated or estimated) or estimated GFR (mL/min/1.73m2) >30% of predicted normal for age.

    Normal GFR by Age : Mean GFR +- SD (mL/min/1.73m^2) 1 week : 40.6 + / - 14.8 2-8 weeks : 65.8 + / - 24.8 >8 weeks : 95.7 + / - 21.7 2-12 years : 133.0 + / - 27.0 13-21 years (males) : 140.0 + / - 30.0 13-21 years (females) : 126.0 + / - 22.0

    GFR, glomerular filtration rate; SD, standard deviation; Greater than 2 years old: Normal GFR is 100 mL/ min; Infants: GFR must be corrected for body surface area.

    Exclusion Criteria:

    Exclusion criteria for both cohorts:

    ° 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1: Patients with High-Risk Disease

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 Units
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
Radiation: Total Body Irradiation
Hyper-fractionated TBI is administered by a linear accelerator at a dose rate of <20 cGy/minute. Treatment planning begins with simulation.
Other Names:
  • TBI
Drug: Cyclophosphamide
Cyclophosphamide is an alkylating agent that prevents cell division by cross-linking DNA strands and decreasing DNA synthesis.
Other Names:
  • Cytoxan
  • Neosar
Drug: Fludarabine
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
Other Names:
  • Fludara
Drug: Clofarabine
Clofarabine, a purine (deoxyadenosine) nucleoside analog, is metabolized to clofarabine 5'-triphosphate.
Other Names:
  • Clolar
Drug: Busulfan
Busulfan is a bifunctional alkylating agent known chemically as 1,4- butanediol, dimethanesulfonate.
Other Names:
  • Busulfex
Drug: Thiotepa
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.
Other Names:
  • Thioplex
Drug: Tacrolimus
Tacrolimus inhibits T-lymphocyte activation
Other Names:
  • Prograf
Drug: Mycophenolate Mofetil
Mycophenolate exhibits a cytostatic effect on T and B lymphocytes.
Other Names:
  • CellCept
Drug: Cyclosporine
Cyclosporine is a calcineurin inhibitor that inhibits production and release of interleukin II and inhibits interleukin II-induced activation of resting T-lymphocytes.
Experimental: Cohort 2: Patients with Very High-Risk Disease

  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 Units
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
Radiation: Total Body Irradiation
Hyper-fractionated TBI is administered by a linear accelerator at a dose rate of <20 cGy/minute. Treatment planning begins with simulation.
Other Names:
  • TBI
Drug: Cyclophosphamide
Cyclophosphamide is an alkylating agent that prevents cell division by cross-linking DNA strands and decreasing DNA synthesis.
Other Names:
  • Cytoxan
  • Neosar
Drug: Fludarabine
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
Other Names:
  • Fludara
Drug: Clofarabine
Clofarabine, a purine (deoxyadenosine) nucleoside analog, is metabolized to clofarabine 5'-triphosphate.
Other Names:
  • Clolar
Drug: Busulfan
Busulfan is a bifunctional alkylating agent known chemically as 1,4- butanediol, dimethanesulfonate.
Other Names:
  • Busulfex
Drug: Thiotepa
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.
Other Names:
  • Thioplex
Drug: Tacrolimus
Tacrolimus inhibits T-lymphocyte activation
Other Names:
  • Prograf
Drug: Mycophenolate Mofetil
Mycophenolate exhibits a cytostatic effect on T and B lymphocytes.
Other Names:
  • CellCept
Drug: Cyclosporine
Cyclosporine is a calcineurin inhibitor that inhibits production and release of interleukin II and inhibits interleukin II-induced activation of resting T-lymphocytes.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Disease-free Survival (DFS)
Time Frame: 1 year
Disease-free Survival (DFS) at 1 year after CBT
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Memorial Sloan Kettering Cancer Center

Investigators

  • Principal Investigator: Andromachi Scaradavou, MD, Memorial Sloan Kettering Cancer Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 28, 2026

Primary Completion (Estimated)

April 28, 2030

Study Completion (Estimated)

April 28, 2030

Study Registration Dates

First Submitted

April 29, 2026

First Submitted That Met QC Criteria

April 29, 2026

First Posted (Actual)

May 5, 2026

Study Record Updates

Last Update Posted (Actual)

May 14, 2026

Last Update Submitted That Met QC Criteria

May 11, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 26-168

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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