Treatment Clinical Trials for Childhood Acute Myeloid Leukemia

Clinical trials are research studies that involve people. The clinical trials on this list are for childhood acute myeloid leukemia treatment. All trials on the list are supported by NCI.

NCI’s basic information about clinical trials explains the types and phases of trials and how they are carried out. Clinical trials look at new ways to prevent, detect, or treat disease. You may want to think about taking part in a clinical trial. Talk to your doctor for help in deciding if one is right for you.

Trials 1-20 of 20
  • A Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients With Relapsed or Refractory Malignancies

    An open-label, global, multi-center study to evaluate the safety and pharmacokinetics of venetoclax monotherapy, to determine the dose limiting toxicity (DLT) and the recommended Phase 2 dose (RPTD), and to assess the preliminary efficacy of venetoclax in pediatric and young adult participants with relapsed or refractory malignancies.
    Location: 8 locations

  • Donor-Derived Tumor-Associated Antigen-Specific T Cells in Treating Participants with Relapsed or Refractory acute myeloid leukemia or myelodysplastic syndrome

    This phase I trial studies the best dose and how well donor-derived multi-tumor-associated antigen specific T cells work in treating participants with acute myeloid leukemia or myelodysplastic syndrome that have come back or does not respond. Tumor associated antigen-specific T cells are immune system cells that may target cell proteins specific to tumor cells.
    Location: 3 locations

  • Donor Stem Cell Transplant in Treating Patients With Hematological Malignancies Who Have Undergone Donor Stem Cell Transplant

    In an effort to lower the occurrences and severity of graft-versus-host disease in patients and to lower the rate of transplant failure, researchers would like to specially treat the donor's blood cells to remove cells that are most likely to attack patients' tissues. This will occur in combination with intense conditioning treatment that patients will receive before having a transplant.
    Location: 3 locations

  • T-allo10 Cell Infusion before Donor Stem Cell Transplant in Treating Patients with Relapsed or Refractory Blood Cancer

    This phase I trial studies side effects and best dose of T-allo10 cells and to see how well they work when given before donor stem cell transplant in treating patients with blood cancer that has come back or does not respond to treatment. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft versus host disease). Giving T-allo10 cells before the transplant may help prevent this from happening. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient’s immune cells and help destroy any remaining cancer cells. Giving T-allo10 cells before donor stem cell transplant may work better in treating patients with blood cancer that has come back or dose not respond to treatment.
    Location: 2 locations

  • Selective Depletion of CD45RA+ T Cells from Allogeneic Peripheral Blood Stem Cell Grafts from HLA-Matched Related and Unrelated Donors in Preventing GVHD

    This phase II trial is for patients with acute lymphocytic leukemia, acute myeloid leukemia, myelodysplastic syndrome or chronic myeloid leukemia who have been referred for a peripheral blood stem cell transplantation to treat their cancer. In these transplants, chemotherapy and total-body radiotherapy ('conditioning') are used to kill residual leukemia cells and the patient's normal blood cells, especially immune cells that could reject the donor cells. Following the chemo / radiotherapy, blood stem cells from the donor are infused. These stem cells will grow and eventually replace the patient's original blood system, including red cells that carry oxygen to our tissues, platelets that stop bleeding from damaged vessels, and multiple types of immune-system white blood cells that fight infections. Mature donor immune cells, especially a type of immune cell called T lymphocytes (or T cells) are transferred along with these blood-forming stem cells. T cells are a major part of the curative power of transplantation because they can attack leukemia cells that have survived the chemo / radiation therapy and also help to fight infections after transplantation. However, donor T cells can also attack a patient's healthy tissues in an often-dangerous condition known as Graft-Versus-Host-Disease (GVHD). Drugs that suppress immune cells are used to decrease the severity of GVHD; however, they are incompletely effective and prolonged immunosuppression used to prevent and treat GVHD significantly increases the risk of serious infections. Removing all donor T cells from the transplant graft can prevent GVHD, but doing so also profoundly delays infection-fighting immune reconstitution and eliminates the possibility that donor immune cells will kill residual leukemia cells. Work in animal models found that depleting a type of T cell, called naive T cells or T cells that have never responded to an infection, can diminish GVHD while at least in part preserving some of the benefits of donor T cells including resistance to infection and the ability to kill leukemia cells. This clinical trial studies how well the selective removal of naive T cells works in preventing GVHD after peripheral blood stem cell transplants. This study will include patients conditioned with high or medium intensity chemo / radiotherapy who can receive donor grafts from related or unrelated donors.
    Location: 2 locations

  • Pevonedistat and Decitabine in Treating Patients with High Risk Acute Myeloid Leukemia

    This phase I trial studies the side effects and best dose of pevonedistat when given together with decitabine in treating patients with high risk acute myeloid leukemia. Pevonedistat and decitabine may stop the growth of cancer cells by blocking some of the enzymes need for cell growth.
    Location: City of Hope Comprehensive Cancer Center, Duarte, California

  • Low-Dose Daunorubicin Hydrochloride in Treating Patients with Relapsed or Refractory Acute Myeloid Leukemia or Acute Lymphoblastic Leukemia

    This phase I trial studies how well low-dose daunorubicin hydrochloride works in treating patients with acute myeloid leukemia or acute lymphoblastic leukemia that has come back or has not responded to treatment. Drugs used in chemotherapy, such as daunorubicin hydrochloride, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
    Location: University of Kansas Cancer Center, Kansas City, Kansas

  • Blinatumomab and T Cell Depleted Donor Blood Cell Transplant in Treating Younger Patients with Relapsed or Refractory Hematologic Malignancy after a Previous Transplant

    This phase II trial studies how well blinatumomab and T cell depleted donor blood cell transplant work in treating children and young adults with hematologic cancer that has not responded or has come back after a previous transplant. White blood cells from donors may be able to kill cancer cells in patients with hematologic cancer. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Removing the T cells from the donor cells before the transplant may stop this from happening. Monoclonal antibodies, such as blinatumomab, may interfere with the ability of cancer cells to grow and spread. Giving blinatumomab after a blood cell transplant may destroy any remaining cancer cells.
    Location: St. Jude Children's Research Hospital, Memphis, Tennessee

  • Fludarabine, Cyclophosphamide, and Total-Body Irradiation before Donor Stem Cell Transplant in Treating Patients with Blood Diseases

    This phase II trial studies how well fludarabine, cyclophosphamide, and total-body irradiation before donor stem cell transplant work in treating patients with blood diseases. Giving chemotherapy, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient’s immune cells and help destroy any remaining cancer cells.
    Location: University of Minnesota / Masonic Cancer Center, Minneapolis, Minnesota

  • High Throughput Drug Sensitivity Assay and Genomics- Guided Treatment of Patients with Relapsed or Refractory Acute Leukemia

    This pilot clinical trial studies the feasibility of choosing treatment based on a high throughput ex vivo drug sensitivity assay in combination with mutation analysis for patients with acute leukemia that has returned after a period of improvement or does not respond to treatment. A high throughput screening assay tests many different drugs individually or in combination that kill leukemia cells in tiny chambers at the same time. High throughput drug sensitivity assay and mutation analysis may help guide the choice most effective for an individual’s acute leukemia.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • Genetically Modified Donor Stem Cell Transplant Followed by Zoledronic Acid in Treating Younger Patients with Relapsed / Refractory Hematologic Malignancies or High Risk Solid Tumors

    This phase I trial studies the side effects of zoledronic acid given after genetically modified donor stem cell transplant in treating younger patients with hematologic malignancies or high risk tumors that have returned after a period of improvement (relapsed) or do not respond to treatment (refractory). Giving chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When healthy stem cells from a donor that have been genetically modified are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving mycophenolate mofetil and tacrolimus after the transplant may stop this from happening. Giving zoledronic acid after the transplant may help strengthen the immune system and make the immune cells work better.
    Location: University of Wisconsin Hospital and Clinics, Madison, Wisconsin

  • Intra-osseous Donor Umbilical Cord Blood and Mesenchymal Stromal Cell Co-transplant in Treating Patients with Hematologic Malignancies

    This clinical trial studies intra-osseous donor umbilical cord blood and mesenchymal stromal cell co-transplant in treating patients with hematologic malignancies. Giving low doses of chemotherapy and total-body irradiation before a co-transplant of donor umbilical cord blood and mesenchymal stromal cells into the bone (intra-osseous) helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil at the time of transplant may stop this from happening.
    Location: Case Comprehensive Cancer Center, Cleveland, Ohio

  • CD34+ Stem Cell Selection in Preventing Graft-Versus-Host Disease in Younger Patients with Malignant Disease Undergoing Donor Stem Cell Transplant

    This phase I / II trial studies cluster of differentiation 34 positive (CD34+) stem cell selection in preventing graft-versus-host-disease (GVHD) in younger patients with malignant disease undergoing donor stem cell transplant. Selected CD34+ stem cell may help stop the patient's immune system from rejecting the donor's stem cells and prevent GVHD.
    Location: NYP / Columbia University Medical Center / Herbert Irving Comprehensive Cancer Center, New York, New York

  • Donor Progenitor Cell and Natural Kill Cell Transplant in Treating Younger Patients with High-Risk Hematologic Malignancies

    This phase II trial studies how well donor progenitor cell and natural killer cell transplant works in treating younger patients with cancers of the blood that are at high risk of coming back or spreading. Giving chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient’s immune system from rejecting the donor’s stem cells. When certain stem cells and natural killer cells from a donor are infused into the patient they may help the patient’s bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body’s normal cells. Removing the T cells from the donor cells before transplant may stop this from happening.
    Location: St. Jude Children's Research Hospital, Memphis, Tennessee

  • Reduced-Intensity Conditioning before Donor Stem Cell Transplant in Treating Patients with High-Risk Hematologic Malignancies

    This phase II trial studies reduced-intensity conditioning before donor stem cell transplant in treating patients with high-risk hematologic malignancies. Giving low-doses of chemotherapy and total-body irradiation before a donor stem cell transplant helps stop the growth of cancer cells. It may also stop the patient’s immune system from rejecting the donor’s stem cells. The donated stem cells may replace the patient’s immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Giving an infusion of the donor’s T cells (donor lymphocyte infusion) before the transplant may help increase this effect.
    Location: Thomas Jefferson University Hospital, Philadelphia, Pennsylvania

  • Conventional and Regulatory T Cells in Treating Patients with Advanced Hematologic Malignancies Undergoing T Cell-Depleted Donor Stem Cell Transplant

    This phase I / II trial studies the side effects and best dose of conventional T cells and regulatory T cells and to see how well they work in treating patients with hematologic malignancies that have spread to other places in the body and are undergoing T cell-depleted donor stem cell transplant. Giving chemotherapy and total body irradiation before a donor stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Removing the T cells from the donor cells before transplant may stop this from happening. Giving an infusion of the donor's T cells (donor lymphocyte infusion) later may help the patient's immune system see any remaining cancer cells as not belonging in the patient's body and destroy them (called graft-versus-tumor effect).
    Location: Stanford Cancer Institute Palo Alto, Palo Alto, California

  • Fludarabine Phosphate, Busulfan, Anti-thymocyte Globulin, and Total-Body Irradiation before Donor Stem Cell Transplant in Treating Younger Patients with Leukemia

    This clinical trial studies fludarabine phosphate, busulfan, anti-thymocyte globulin, and total-body irradiation before donor stem cell transplantation in treating younger patients with leukemia. Giving chemotherapy and total-body irradiation before a donor stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets.
    Location: Lurie Children's Hospital-Chicago, Chicago, Illinois

  • Fludarabine, Cyclophosphamide, and Total-Body Irradiation in Treating Patients Who Are Undergoing an Umbilical Cord Blood Transplant for Hematologic Cancer

    This phase II trial studies how well giving fludarabine phosphate and cyclophosphamide together with total-body irradiation works in treating patients who are undergoing an umbilical cord blood transplant for hematologic cancer. Giving chemotherapy drugs, such as fludarabine phosphate and cyclophosphamide, and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells and prepares the patient's bone marrow for the stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil may stop this from happening.
    Location: University of Minnesota / Masonic Cancer Center, Minneapolis, Minnesota

  • KIR Favorable Mismatched Haplo Transplant and KIR Polymorphism in ALL / AML / MDS Allo-HCT Children

    This is a phase II, open-label, non-randomized, prospective study of haploidentical transplantation using KIR-favorable donors for children with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT). The relationship of KIR2DL1 polymorphisms to survival in children with these diseases undergoing any approach to allogeneic HCT during the study time frame will also be determined.
    Location: 6 locations

  • Daratumumab and Donor Lymphocyte Infusion in Treating Participants with Relapsed Acute Myeloid Leukemia after Stem Cell Transplant

    This phase I / II trial studies the side effects and best dose of donor lymphocyte infusions when given together with daratumumab and to see how well they work in treating participants with acute myeloid leukemia that has come back after a stem cell transplant. A donor lymphocyte infusion is a type of therapy in which lymphocytes (white blood cells) from the blood of a donor are given to a participant who has already received a stem cell transplant from the same donor. The donor lymphocytes may kill remaining cancer cells. Monoclonal antibodies, such as daratumumab, may interfere with the ability of cancer cells to grow and spread. Giving daratumumab and donor white blood cells may work better in treating participants with acute myeloid leukemia.
    Location: Ohio State University Comprehensive Cancer Center, Columbus, Ohio