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.
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.
Sponsors
lead: Memorial Sloan Kettering Cancer Center

