A Phase I/II Study of ECT-001-Expanded Cord Blood Transplants in Young People with Bone Marrow Cancers


Full Title

A Phase I/II Open-Label Study of ECT-001-Expanded Single Unit Cord Blood Transplantation in Pediatric and Young Adult (<21 Years) Patients with High Risk and Very High- Risk Myeloid Malignancies.


Blood from the umbilical cord and placenta donated by a newborn’s mother is rich in healthy “stem” cells that can fight cancer. Cord blood transplants are a standard treatment for children and young adults with bone marrow cancers. With a cord blood transplant, however, it can take longer for blood cell counts to recover than in patients who receive a bone marrow or stem cell transplant. Low blood cell counts can lead to bleeding or infection. The cord blood must also be a good match, and the number of cells in cord blood must high enough. On the other hand, data also suggest that the chance of experiencing a relapse is likely lower in patients who receive a cord blood transplant, especially those who have a high risk of relapse to being with. 

Researchers think that increasing the number of stem cells in cord blood using a process called “expansion” will help blood cell counts recover faster. It may also reduce complications after a cord blood transplant. This study is evaluating a new expansion approach. The technology uses a molecule called UM171 and a process that increases the number of stem cells in cord blood. UM171 helps promote the growth of stem cells. A study in adults showed that this product is safe and effective for controlling leukemia in patients with a very high risk of relapse.

Cord blood that has been expanded using this technology is called “ECT-001-expanded cord blood.” If this expansion approach is safe and effective, it will allow doctors to use donated cord blood that does not have a high number of cells to treat bone marrow cancers. In this study, researchers will see if a cord blood transplant using ECT-001-expanded cord blood is safe, practical, and effective in patients under age 21 with different types of bone marrow cancer.

Who Can Join

To be eligible for this study, patients must meet several requirements, including:

  • Participants must have a bone marrow cancer that continues to grow despite treatment. Examples include acute myeloid leukemia, myelodysplastic syndrome, chronic myelogenous leukemia, mixed-phenotype acute leukemia, and juvenile myelo-monocytic leukemia.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for children, adolescents, and young adults under age 21.

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




Phase II (phase 2)

Disease Status

Relapsed or Refractory


ClinicalTrials.gov ID