Actively Recruiting

Phase 2
Age: 0 - 21Years
All Genders
NCT04644016

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

Led by Memorial Sloan Kettering Cancer Center · Updated on 2026-03-13

31

Participants Needed

1

Research Sites

317 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.

CONDITIONS

Official Title

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

Who Can Participate

Age: 0 - 21Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients aged 21 years or younger at time of consent with no available matched related or unrelated donor
  • Diagnosed with AML, ALL, other acute leukemias, MDS or MPD without myelofibrosis, NHL or HL at high risk, inherited metabolic disorders, hemoglobinopathies, bone marrow failure, or HLH
  • AML patients must meet specific remission and risk criteria as detailed in the protocol
  • ALL patients must meet specific remission and risk criteria as detailed in the protocol
  • Other acute leukemias with less than 5% blasts by bone marrow morphology
  • MDS/MPD patients with specific risk scores and bone marrow counts as detailed
  • NHL or HL patients with specified remission or progression status
  • For metabolic diseases, early disease status before neurologic symptoms
  • Karnofsky or Lansky score of 70% or higher
  • Bilirubin level 1.5 mg/dL or less (unless benign congenital hyperbilirubinemia)
  • ALT level 3 times upper limit of normal or less
  • Pulmonary function tests at or above 50% predicted
  • Left ventricular ejection fraction 50% or higher
  • Age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index (aaHCT-CI) of 7 or less
  • Renal function with serum creatinine 1.5 times normal or less or adequate creatinine clearance or glomerular filtration rate
  • Cord blood graft meeting MSKCC selection criteria with appropriate HLA matching and cell dose
Not Eligible

You will not qualify if you...

  • Inadequate performance status or organ function
  • Advanced metabolic disease
  • Active central nervous system leukemic involvement
  • Indolent NHL or HL with disease progression after most recent chemotherapy
  • Diagnosis of myelofibrosis or malignancy with moderate to severe bone marrow fibrosis
  • Autologous stem cell transplant within 6 months before enrollment
  • Any prior allogeneic stem cell transplant
  • Active and uncontrolled infections at time of transplantation
  • HIV infection
  • Seropositivity for HTLV-1
  • Pregnancy or breastfeeding
  • Inability of patient or guardian to provide informed consent or comply with treatment protocol and follow-up

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Memorial Sloan Kettering Cancer Center

New York, New York, United States, 10065

Actively Recruiting

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Research Team

M

Maria I Cancio, MD

CONTACT

J

Jaap Jan Boelens, MD, PhD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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