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Allogeneic (cells from a donor) blood or marrow transplant (BMT) using umbilical cord blood to treat blood cancers and blood disorders in children, teens and young adults

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21 and younger

Phase 2

1 Location

NCT04644016

Clinical Trial Goal


To find out if allogeneic BMT using umbilical cord blood is safe and works well to treat blood cancers and blood disorders in children, teens and young adults

You may be able to join this trial if you:


Your child:
  • Is 21 years old or younger
  • Has been diagnosed with a blood cancer or blood disorder. Some examples include: 
    • Acute lymphoblastic leukemia (ALL)
    • Acute myeloid leukemia (AML)
    • Blastic plasmacytoid dendritic cell neoplasm (BPDCN)
    • Bone marrow failure syndromes (such as Diamon Blackfan anemia
    • Immunodeficiencies (such as hemophagocytic lymphohistiocytosis (HLH))
    • Myelodysplastic syndrome (MDS)
    • Myeloproliferative disorder (MPD)
    • Lymphoma (Hodgkin or non-Hodgkin)
    • Metabolic disorders (such as ALD, Hurler syndrome, Krabbe disease, or MLD)
  • Does not have a matched donor available. Your doctor can tell you this
  • Does not have myelofibrosis
  • Has not had an autologous (your own cells) blood or marrow transplant (BMT) in the last 6 months
  • Has not had an allogeneic BMT
You agree to have other standard tests done to see if your child can be in the clinical trial 

Trial Details


Busulfan, clofarabine and fludarabine are chemotherapy (chemo) drugs that block the growth of cancer cells.
Cyclosporine and mycophenolate mofetil are drugs that slow down the immune system.

Before transplant, your child will get standard treatment with the following:
  • Busulfan – Given as intravenous (IV) infusions 
  • Clofarabine - Given as IV infusions 
  • Cyclosporine - A pill that you take by mouth 
  • Fludarabine - Given as IV infusions
  • Mycophenolate mofetil - A pill that you take by mouth 

On transplant day, the blood-forming cells from umbilical cord blood are given to them through an IV infusion.

After transplant, your child may continue on cyclosporine and mycophenolate mofetil.

Your child may continue treatment for as long as the clinical trial doctors think it’s best for their health. Your child will have biopsies and/or scans to see how well the treatment is working. The clinical trial doctors will check your child's health for 1 year.  

The Food and Drug Administration (FDA) has approved allogeneic BMT using umbilical cord blood and all of the drugs used in this trial. 

Contacts


Maria I Cancio, MD, 212-639-2446, canciom@mskcc.org

Jaap Jan Boelens, MD, PhD, 212-639-3643, boelensj@mskcc.org

Locations


Memorial Sloan Kettering Cancer CenterRECRUITING

New York, New York
Contacts:

  • Maria Cancio, MD, 212-639-2446
  • E.

ClinicalTrials.gov record


NCT04644016. First posted on 11/25/20

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