A Study of Cord Blood Transplantation in Children and Young Adults with Blood Cancers and Non-Cancerous Blood Disorders


Full Title

Cord Blood Transplantation in Children and Young Adults with Hematologic Malignancies and Non-Malignant Disorders


The transplantation of stem cells from umbilical cord blood is a treatment for some blood cancers and non-cancerous blood or metabolic disorders. Patients routinely receive high doses of chemotherapy and sometimes radiation before receiving the stem cells to help make room in the bone marrow for new blood stem cells to grow, prevent the body from rejecting the transplanted cells, and help kill any abnormal blood cells in the body. However, the combination of these treatments can have serious side effects.

Researchers are doing this study to find out whether a combination of the chemotherapy drugs clofarabine, fludarabine, and busulfan (without radiation) is a safe and effective treatment for children and young adults receiving cord blood transplants for blood cancers or non-cancerous blood or metabolic disorders. These three drugs are given intravenously (by vein).

Who Can Join

To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have a blood cancer or non-cancerous blood disorder and be candidates for umbilical cord transplantation. Examples of blood cancers include acute myelogenous leukemia (AML), acute lymphoblastic leukemia (ALL), advanced myelodysplasia (MDS), chronic myelogenous leukemia (CML), myeloproliferative disorders, and non-Hodgkin and Hodgkin lymphoma. Examples of non-cancerous disorders include inherited metabolic diseases, Hurler syndrome, Hunter disease, Sly syndrome, alpha-mannosidosis, X-ALD (X-linked adrenoleukodystrophy), osteopetrosis, metachromatic leukodystrophy, globoid cell leukodystrophy, bone marrow failure syndromes, and diseases like HLH (hemophagocytic lymphohistiocytosis) caused by a compromised immune system.
  • This study is for patients age 21 and younger.

For more information about this study and to inquire about eligibility, please contact 1-833-MSK-KIDS.




Phase II (phase 2)

Disease Status

Relapsed or Refractory


ClinicalTrials.gov ID