Acute Myelogenous LeukemiaPhase 2NCT07566377
Cord Blood Transplantation in Children and Young Adults With Blood Cancer
A Phase 2 study testing Cord Blood Units, Total Body Irradiation and Cyclophosphamide in people with Acute Myelogenous Leukemia and Acute Lymphoblastic Leukemia, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 26 years
- Conditions the study is for
- Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes
- What is being tested?
- Cord Blood Units, Total Body Irradiation and Cyclophosphamide
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 71 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Apr 2026to 28 Apr 2030
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 26 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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:
- 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)
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- 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.
- 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.
- 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
- 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)
- 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:
- 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.
- 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.
Who cannot join
- ° 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Memorial Sloan Kettering Cancer Center (All Protocol Activities)
New York, New York, 10065
Andromachi Scaradavou, MD, 1-833-MSK-KIDS
About this study
Official title: 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.
- Conditions
- Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Non-hodgkin Lymphoma, Hodgkin Lymphoma, Leukemia
- Being tested
- Cord Blood Units, Total Body Irradiation, Cyclophosphamide, Fludarabine, Clofarabine
- Keywords
- Cord blood transplants, Graft-versus-host Disease, Lymphoma, Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Myelodysplastic Syndromes, Acute Lymphoblastic Leukemia, Acute Myelogenous Leukemia, Memorial Sloan Kettering Cancer Center, 26-168
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Lymphoma, Non-Hodgkin, Hodgkin Disease, Leukemia, Lymphoma, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cord Blood UnitsBiological
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
Total Body IrradiationRadiation
Also called TBI
Hyper-fractionated TBI is administered by a linear accelerator at a dose rate of <20 cGy/minute. Treatment planning begins with simulation.
CyclophosphamideDrug
Also called Cytoxan, Neosar
Cyclophosphamide is an alkylating agent that prevents cell division by cross-linking DNA strands and decreasing DNA synthesis.
FludarabineDrug
Also called Fludara
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
ClofarabineDrug
Also called Clolar
Clofarabine, a purine (deoxyadenosine) nucleoside analog, is metabolized to clofarabine 5'-triphosphate.
BusulfanDrug
Also called Busulfex
Busulfan is a bifunctional alkylating agent known chemically as 1,4- butanediol, dimethanesulfonate.
ThiotepaDrug
Also called Thioplex
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.
TacrolimusDrug
Tacrolimus inhibits T-lymphocyte activation
Mycophenolate exhibits a cytostatic effect on T and B lymphocytes.
CyclosporineDrug
Cyclosporine is a calcineurin inhibitor that inhibits production and release of interleukin II and inhibits interleukin II-induced activation of resting T-lymphocytes.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Patients with High-Risk Disease | 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). Receives: Biological: Cord Blood Units; Radiation: Total Body Irradiation; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Clofarabine; Drug: Busulfan; Drug: Thiotepa; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Drug: Cyclosporine |
| Cohort 2: Patients with Very High-Risk Disease | 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. Receives: Biological: Cord Blood Units; Radiation: Total Body Irradiation; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Clofarabine; Drug: Busulfan; Drug: Thiotepa; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Drug: Cyclosporine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease-free Survival (DFS)
Disease-free Survival (DFS) at 1 year after CBT
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
28 Apr 202628 Apr 2030
- First posted5 May 2026
- Study start28 Apr 2026
- Primary completion28 Apr 2030 (estimated)
- Study completion28 Apr 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myelogenous LeukemiaStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 71 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Andromachi Scaradavou, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07566377
- Sponsor's protocol ID
- 26-168
- Status last verified
- May 2026
- First submitted
- 29 Apr 2026
- Last updated
- 14 May 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.