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Cord blood transplant for children and young adults with blood cancers.

Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies

Phase 2 Interventional Memorial Sloan Kettering Cancer Center · NCT07566377

This study will test whether a cord blood transplant given as a first or second transplant can help children and young adults with leukemia, myelodysplasia, or lymphoma who lack a matched donor.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment71 (estimated)
AgesN/A to 26 Years
SexAll
SponsorMemorial Sloan Kettering Cancer Center Academic / other
Drugs / interventionsblinatumomab, inotuzumab, CAR T, CAR-T, chemotherapy
Locations7 sites (Basking Ridge, New Jersey and 6 other locations)
Trial IDNCT07566377 on ClinicalTrials.gov

What this trial studies

This is a phase 2 interventional trial using unrelated cord blood units for pediatric and young adult patients with high-risk or relapsed hematologic malignancies. Patients receive a preparative regimen that may include total body irradiation and chemotherapy agents such as cyclophosphamide, fludarabine, or clofarabine before cord blood infusion. Eligible participants are age 26 or younger and must not have a suitably matched related or unrelated donor available within four weeks. Outcomes of interest include engraftment, graft-versus-host disease, disease control, and survival after transplant.

Who should consider this trial

Good fit: Children and young adults (age ≤26) with high-risk or relapsed acute leukemias, myelodysplastic syndromes, or lymphoma who do not have a suitably matched related or unrelated donor available within four weeks are the intended candidates.

Not a fit: Patients who have an available suitably matched donor, are older than the age limit, or have medical conditions that make transplant unsafe are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, this approach could provide an effective transplant option for young patients who lack a matched donor, potentially improving disease control and long-term survival.

How similar studies have performed: Cord blood transplantation is an established option for patients without matched donors and has cured some patients, but outcomes and risks such as graft-versus-host disease vary, so further data in this young high-risk group are needed.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Basking Ridge, New Jersey and 6 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Acute Myelogenous LeukemiaAcute Lymphoblastic LeukemiaMyelodysplastic SyndromesNon-hodgkin LymphomaHodgkin LymphomaLeukemiaLymphomaGraft-versus-host Disease
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.