A Phase I/II Study of SMART101 in Children and Adults with Acute Leukemia Who Receive a T-Cell Depleted Stem Cell Transplant from a Donor


Full Title

A phase I/II Study Evaluating the Safety and the Efficacy of SMART101 Human T Lymphoid Progenitor (HTLP) Injection to Accelerate Immune Reconstitution after T cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric and Adult Patients with Acute Leukemia Hematological Malignancies


The purpose of this study is to assess the safety and effectiveness of SMART101, a new cellular therapy for reducing the risk of infection by speeding the rebuilding of the immune system after allogeneic bone marrow transplantation (the receipt of stem cells from a donor). SMART101 is being evaluated in children and adults with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) that is difficult to treat using standard therapies.

During this study, participants will receive healthy blood-forming cells from a donor to replace the diseased bone marrow. The type of transplant they will receive is called a T-cell depleted transplant. T-cell depleted transplants are used to prevent a serious side effect called graft-versus-host disease (GvHD), which occurs when a donor’s T cells (white blood cells) attack the cells of the recipient’s body. However, T-cell depleted transplants can lead to severe infections due to a low number of donated T cells.

Researchers think SMART101 may reduce the risk of infection by speeding the production of donor T cells after a T-cell depleted transplant. SMART101 is made in a laboratory using a procedure that generates large numbers of “precursor” T cells from the donor. These cells cannot cause GvHD because they mature in the body of the transplant recipient, but they can potentially reduce infection. SMART101 is given intravenously (by vein) 4-10 days after the transplant.

Who Can Join

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

  • Participants must have high-risk AML or ALL and be candidates for a T-cell depleted stem cell transplant from a donor.
  • Patients must be physically well enough that they are able to be mobile, take care of themselves, and engage in all but physically strenuous activities. For example, they must be well enough that they could carry out office work or light housework.
  • This study is for people of all ages.

For more information and to ask about eligibility for this study, please contact the office of Dr. Jaap Jan Boelens at 212-639-3643.




Phase I/II (phases 1 and 2 combined)


ClinicalTrials.gov ID