Cord Blood Transplant for Blood Cancers and Non-malignant Disorders
This study is looking at a cord blood transplant approach for children and young adults up to 21 years old who have certain blood cancers like AML (Acute Myelogenous Leukemia), ALL (Acute Lymphoblastic Leukemia), or other blood disorders, and don't have a suitable matched donor. The transplant uses a combination of medicines: Clofarabine, Fludarabine, Busulfan, Cyclosporine-A, and Mycophenolate Mofetil. Researchers want to see how many patients experience treatment-related mortality (death due to the treatment) within one year after the transplant. This study aims to enroll 31 participants.
- Study design
- This is a single-arm study, meaning all participants receive the same treatment. It plans to enroll 31 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for one year after the cord blood transplant to assess treatment-related mortality.
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NCT04644016
Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
Recruiting
PHASE2Up to 21InterventionalTreatmentMemorial Sloan Kettering Cancer CenterInvestigator-initiated
~31 participants
Updated 2026-03-13 on ClinicalTrials.gov
What's tested:ClofarabineFludarabineBusulfanCyclosporine-AMycophenolate MofetilCord Blood Graft
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Treatment related mortality at 1 year after myeloablative cord transplant
Measured over 1 year
Conditions
Where it's being run
1 sites across 1 statesNew York1
Study leadership
- Maria Cancio, MD · PRINCIPAL_INVESTIGATOR · Memorial Sloan Kettering Cancer Center
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
Complete first remission (CR1) at high risk for relapse such as any of the following:
Known prior diagnosis of myelodysplasia (MDS) or myeloproliferative disorder (MPS).
Therapy-related AML (t-AML).
White cell count at presentation \> 100,000.
Presence of extramedullary leukemia at diagnosis.
Any unfavorable subtype by FAB or WHO classification.
High-risk cytogenetics (e.g. those associated with MDS, abnormalities of 5, 7, 8, complex karyotype) or high-risk molecular abnormalities.
Requirement for 2 or more inductions to achieve CR1.
Presence of Minimal Residual Disease (MRD+) by cytogenetics, flow cytometry or molecular methods after induction.
Any patient with newly diagnosed AML with intermediate risk cytogenetics who elects allograft with curative intent over consolidation chemotherapy.
Any patient unable to tolerate consolidation chemotherapy as would have been deemed appropriate by the treating physician.
Other high-risk features not defined above.
Complete second remission (CR2).
Primary refractory or relapsed AML with less than 10% blasts by bone marrow morphology. Patients with cytogenetic, flow cytometric, or molecular abnormalities in ≤ 10% of cells are eligible.
Complete first remission (CR1) at high risk for relapse such as any of the following:
White cell count at presentation \> 30,000 for B-cell lineage and \> 100,000 for T-cell lineage.
Presence of any high-risk cytogenetic abnormalities such as t (9;22), t (1;19), t (4;11) or other MLL rearrangements (11q23) or other high-risk molecular abnormality.
Failure to achieve complete remission (CR) after four weeks of induction therapy.
Persistence or recurrence of MRD on therapy.
Any patient unable to tolerate consolidation and/or maintenance chemotherapy as would have been deemed appropriate by the treating physician.
Other high-risk features not defined above.
Complete second remission (CR2).
Primary refractory or relapsed ALL with MRD disease after antibody therapy (e.g., blinatumomab, inotuzumab, other) and/or CAR-T cell therapy.
International prognostic scoring system (IPSS) risk score of INT-2 or high risk at the time of diagnosis.
Any IPSS risk category if life-threatening cytopenia(s) exists.
Any IPSS risk category with karyotype or genomic changes that indicate high risk for progression to acute myelogenous leukemia.
MDS/ myeloproliferative disorder overlap syndromes without myelofibrosis.
MDS/ MPD patients must have less than 10% bone marrow myeloblasts and ANC ≥ 0.2 (growth factor supported if necessary) at transplant work-up.
Eligible patients with aggressive histology (such as, but not limited to, diffuse large B-cell NHL, mantle cell NHL, and T-cell histology) in CR.
Eligible 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.
Eligible patients with HL will be those without progression of disease (POD) after salvage chemotherapy with no single lesion ≥ 5 cm.
Hurler Syndrome
Hunter (MPS 2 - early disease)
Sly syndrome (MPSVIII)
α-Mannosidosis
X- ALD
Osteopetrosis
Metachromatic Leukodystrophy
Globoid (GLD)
Hemoglobinopathies
Bone Marrow Failure syndromes
Immunodeficiencies, including HLH
Karnofsky or Lansky score ≥ 70% (see Appendix)
Bilirubin ≤ 1.5 mg/dL (unless benign congenital hyperbilirubinemia).
ALT ≤ 3 x upper limit of normal.
Pulmonary function (spirometry and corrected DLCO) ≥ 50% predicted (corrected for hemoglobin) .
Left ventricular ejection fraction ≥ 50%.
Age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index (aaHCT-CI) less than or equal to 7.
Renal: serum creatinine ≤ 1.5x normal for age. If serum creatinine is outside the normal range, then CrCl \> 50 mL/min/1.73m2 (calculated or estimated) or GFR (mL/min/1.72m2) \>30% of predicted normal for age.
Each CB unit must be at least 3/8 HLA-matched to the patient considering high-resolution 8-allele HLA typing.
For malignant diseases follow MSKCC CBU selection algorithm
For non-malignant diseases, CBU will be required to have \> 5 x 107 TNC/kg; high HLA allele level match is preferable
Exclusion
Inadequate performance status/ organ function.
Advanced metabolic disease (EBMT handbook).
Active CNS leukemic involvement.
Indolent NHL or Hodgkin lymphoma with progression of disease after most recent salvage chemotherapy.
Diagnosis of myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis.
Autologous stem cell transplant within the preceding 6 months.
Any prior allogeneic stem cell transplant.
Active and uncontrolled infection (bacterial/fungal/viral) at time of transplantation.
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.
What this trial measures
- Treatment related mortality at 1 year after myeloablative cord transplant1 year
The primary objective of this study is to assess treatment related mortality (TRM) at 1 year after myeloablative cord transplant.