Clinical Trials Using Busulfan

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Clinical trials are research studies that involve people. The clinical trials on this list are studying Busulfan. 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 1-25 of 49
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  • Ruxolitinib Phosphate Before Reduced Intensity Donor Stem Cell Transplant in Treating Patients with Myelofibrosis

    This phase II trial studies how well adding ruxolitinib phosphate before a reduced intensity donor stem cell transplant works in treating patients with myelofibrosis. Ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving ruxolitinib phosphate and chemotherapy before a donor stem cell may help 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.
    Location: 11 locations

  • Chemotherapy before and after Donor Bone Marrow Transplant in Treating Younger Patients with Hematologic Cancer

    This pilot phase II trial studies how well chemotherapy before and after a donor bone marrow transplant works in treating younger patients with hematologic cancer. Giving chemotherapy and total-body irradiation before a donor bone marrow 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 related donor, that do not exactly match the patient’s blood, are infused into the patient they may replace the patient’s immune cells and help destroy any remaining cancer cells. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells called graft-versus-host disease. Giving cyclophosphamide after the transplant may stop this from happening.
    Location: 14 locations

  • HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide

    This is a multi-center, single arm Phase II study of hematopoietic cell transplantation (HCT) using human leukocyte antigen (HLA)-mismatched unrelated bone marrow transplantation donors and post-transplantation cyclophosphamide (PTCy), sirolimus and mycophenolate mofetil (MMF) for graft versus host disease (GVHD) prophylaxis in patients with hematologic malignancies.
    Location: 7 locations

  • Vaccine Therapy after Donor Stem Cell Transplant in Treating Patients with Advanced Myelodysplastic Syndrome or Acute Myeloid Leukemia

    This randomized phase II trial studies how well vaccine therapy after donor stem cell transplant works in treating patients with myelodysplastic syndrome or acute myeloid leukemia that has spread to other places in the body (advanced). Vaccines made from a gene-modified virus and a person's tumor cells may help the body build an immune response to kill cancer cells. Giving chemotherapy before a donor peripheral blood or bone marrow 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. It is not yet known whether giving vaccine therapy after a donor peripheral blood or bone marrow transplant is more effective than transplant alone in treating myelodysplastic syndrome or acute myeloid leukemia.
    Location: 4 locations

  • Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)

    This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care. The primary outcome is 2-year overall survival.
    Location: 3 locations

  • Haploidentical Donor Stem Cell Transplant plus Umbilical Cord Blood Transplant in Treating Patients with Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

    This randomized phase II trial studies how well a partially matched (haploidentical) donor stem cell transplant and umbilical cord blood transplant works compared to a matched unrelated (not a sibling or family member) stem cell donor in treating patients with acute myeloid leukemia or myelodysplastic syndrome that is likely to recur (come back), or spread (high-risk). When matched family members cannot be identified, doctors use the stem cells of other healthy volunteers whose blood type matches the patients' blood. Another transplant procedure uses stem cells from umbilical cord blood (UCB) from the placenta of newborn baby. Chemotherapy and total-body irradiation are given before transplant to help stop the growth of cancer cells in the bone marrow and weaken the immune system so that the transplant cells can grow. It may take time to find a donor who is not a family member but matches the patient's blood type. Giving an umbilical cord blood transplant with a partially matched stem cell transplant may be as effective as a matched stem cell transplant in treating patients with acute myeloid leukemia or high-risk myelodysplastic syndrome.
    Location: 2 locations

  • Pentostatin and Cyclophosphamide, with or without Busulfan before Donor Blood or Bone Marrow Transplant in Treating Patients with Primary Immunodeficiencies

    This pilot phase II trial studies how well pentostatin and cyclophosphamide with or without busulfan before donor blood or bone marrow transplant work in treating patients with decreased body ability to fight infections and other diseases (immunodeficiencies). Giving chemotherapy, such as pentostatin, cyclophosphamide, and busulfan, before a donor blood or bone marrow transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and the disease causing 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 (called graft-versus-host disease). Giving sirolimus, mycophenolate mofetil, and cyclophosphamide after the transplant may stop this from happening.
    Location: 2 locations

  • Immunotherapy Following Reduced Intensity Conditioning and Allogeneic Stem Cell Transplant for Poor Risk CD30+ Hodgkin Lymphoma Patients

    Patients with relapsed or refractory Hodgkin Lymphoma who are CD30+ will receive a standard of care reduced intensity regimen and an allogeneic stem cell transplant (from another person, related or unrelated). Following recovery, patients will receive a medication called Brentuximab Vendotin which is targeted against CD30+ cells. The study hypothesis is that this treatment will be safe and well tolerated in children and young adults.
    Location: 3 locations

  • T-Cell Infusion and Donor Stem Cell Transplant in Treating Patients with High-Risk Hematologic Cancer

    This phase I / II trial studies the side effects and best way to give T-cell infusion and donor stem cell transplant and to see how well it works in treating patients with high-risk hematologic cancer. Giving chemotherapy and total-body irradiation (TBI) before a donor stem cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When certain stem cells from a related donor, that do not exactly match the patient's blood, 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) 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: 2 locations

  • Allogeneic Stem Cell Transplant for Chronic Lymphocytic Leukemia (CLL)

    The goal of this clinical research study is to learn the highest tolerable dose of gemcitabine (out of 4 possible doses) that can be given in combination with busulfan and clofarabine before an allogeneic stem cell transplant. Researchers also want to learn if this combination can help to control CLL. The safety of this treatment will also be studied. Busulfan is designed to bind to DNA (the genetic material of cells), which may cause cancer cells to die. It is commonly used in stem cell transplants. Clofarabine and gemcitabine are designed to block the growth of cancer cells, which may cause the cancer cells to die.
    Location: 2 locations

  • Different Therapies in Treating Infants With Newly Diagnosed Acute Leukemia

    RATIONALE: Giving chemotherapy before a donor stem cell transplant helps stop the growth of cancer cells. It also helps 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. Giving cyclosporine, methotrexate, leucovorin, and antithymocyte globulin before and after transplant may stop this from happening. It is not yet known which treatment regimen is most effective in treating acute leukemia. PURPOSE: This randomized clinical trial is studying how well different therapies work in treating infants with newly diagnosed acute leukemia.
    Location: 2 locations

  • Cyclophosphamide and Bendamustine after Donor Bone Marrow Transplant in Preventing GVHD in Patients with Leukemia or Lymphoma

    This phase I / II trial studies the side effects and best dose of bendamustine when given together with cyclophosphamide after donor bone marrow transplant and to see how well they work in preventing graft versus host disease (GVHD) in patients with leukemia or lymphoma. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving bendamustine and cyclophosphamide after the transplant may stop this from happening.
    Location: The University of Arizona Medical Center-University Campus, Tucson, Arizona

  • Busulfan, Fludarabine Phosphate, and Post-Transplant Cyclophosphamide in Treating Patients with Blood Cancer Undergoing Donor Stem Cell Transplantation

    This phase II trial studies the side effect of busulfan, fludarabine phosphate, and post-transplant cyclophosphamide in treating patients with blood cancer undergoing donor stem cell transplantation. Drugs used in chemotherapy, such as busulfan, fludarabine phosphate and cyclophosphamide 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. Giving chemotherapy such as busulfan and fludarabine phosphate 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving cyclophosphamide after the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them.
    Location: M D Anderson Cancer Center, Houston, Texas

  • Personalized NK Cell Therapy after Chemotherapy and Cord Blood Transplant in Treating Patients with Myelodysplastic Syndrome, Leukemia, Lymphoma or Multiple Myeloma

    This phase II clinical trial studies how well personalized natural killer (NK) cell therapy works after chemotherapy and umbilical cord blood transplant in treating patients with myelodysplastic syndrome, leukemia, lymphoma or multiple myeloma. This clinical trial will test cord blood (CB) selection for human leukocyte antigen (HLA)-C1 / x recipients based on HLA-killer-cell immunoglobulin-like receptor (KIR) typing, and adoptive therapy with CB-derived NK cells for HLA-C2 / C2 patients. Natural killer cells may kill tumor cells that remain in the body after chemotherapy treatment and lessen the risk of graft versus host disease after cord blood transplant.
    Location: M D Anderson Cancer Center, Houston, Texas

  • Lenalidomide and High-Dose Chemotherapy before Stem Cell Transplant in Treating Patients with Relapsed or Refractory, Diffuse Large B-Cell Lymphoma of the ABC Subtype

    This phase I / II trial studies the side effects and best dose of lenalidomide when given together with high-dose chemotherapy (vorinostat, gemcitabine hydrochloride, busulfan, and melphalan) followed by stem cell transplant and to see how well the regimen works in treating patients with diffuse large B-cell lymphoma that has come back (relapsed) or has not responded to treatment (refractory), and is of the activated B-cell-like (ABC) subtype. Lenalidomide may stimulate the immune system to attack cancer cells. Drugs used in chemotherapy, such as vorinostat, gemcitabine hydrochloride, busulfan, and melphalan, 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. Combining lenalidomide with this chemotherapy regimen may be an effective treatment for the ABC subtype of diffuse large B-cell lymphoma.
    Location: M D Anderson Cancer Center, Houston, Texas

  • Panobinostat, Gemcitabine Hydrochloride, Busulfan, and Melphalan before Stem Cell Transplant in Treating Patients with Refractory or Relapsed Multiple Myeloma

    This phase II trial studies how well panobinostat, gemcitabine hydrochloride, busulfan, and melphalan before stem cell transplant work in treating patients with multiple myeloma that does not respond to treatment (refractory) or has returned (relapsed). Panobinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving high-dose chemotherapy, such as gemcitabine hydrochloride, busulfan, and melphalan, before a peripheral blood stem cell transplant helps kill any cancer cells that are in the body and helps make room in the patient’s bone marrow for new blood-forming cells (stem cells) to grow. Previously collected stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.
    Location: M D Anderson Cancer Center, Houston, Texas

  • Donor Hematopoietic Stem Cell Transplant and Combination Chemotherapy as Initial Salvage Therapy in Treating Patients with Acute Myeloid Leukemia Refractory to High-Dose Cytarabine-Based Induction Chemotherapy

    This phase II trial studies the safety of a donor hematopoietic stem cell transplant and combination chemotherapy and to see how well they work as an initial treatment given after cancer has not responded to other treatments (salvage therapy) in treating patients with acute myeloid leukemia that did not respond to treatment (refractory) with high-dose cytarabine-based chemotherapy that was given as the first treatment (induction therapy). Drugs used in chemotherapy, such as decitabine, clofarabine, idarubicin, cytarabine, busulfan, fludarabine phosphate, and anti-thymocyte globulin, 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. Giving more than one drug (combination chemotherapy) may kill more cancer cells. Giving chemotherapy and total-body irradiation before a donor peripheral blood or bone marrow 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. The donated stem cells may also replace the patient’s immune cells and help destroy any remaining cancer cells. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving tacrolimus, mycophenolate mofetil, cyclophosphamide, and methotrexate after the transplant may stop this from happening. Giving a donor hematopoietic stem cell transplant together with combination chemotherapy may be a better treatment for acute myeloid leukemia.
    Location: M D Anderson Cancer Center, Houston, Texas

  • JAK Inhibitor before Donor Stem Cell Transplant in Treating Patients with Primary or Secondary Myelofibrosis

    This phase II trial studies how well giving a JAK inhibitor before a donor stem cell transplant works in treating patients with myelofibrosis that developed without another condition (primary) or evolved from other bone marrow disorders (secondary). JAK inhibitors are a class of drugs that may stop the growth of abnormal cells by blocking an enzyme needed for cell growth. Giving a JAK inhibitor before a donor stem cell transplant may help reduce symptoms of myelofibrosis such as inflammation and enlargement of the spleen, improve the patient’s general physical condition, and prevent complications from occurring after the transplant. Infusing healthy stem cells from a donor into the patient may help the patient's bone marrow work normally and make stem cells, red blood cells, white blood cells, and platelets. Giving a JAK inhibitor before a donor stem cell transplant may help improve transplant outcomes in patients with myelofibrosis.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • Sequential Busulfan, Cladribine, and Fludarabine Phosphate before Donor Stem Cell Transplant in Treating Patients with Acute Myelogenous Leukemia or Myelodysplastic Syndromes

    This randomized phase II trial studies how well sequential busulfan, cladribine, and fludarabine phosphate before donor stem cell transplant work in treating patients with acute myelogenous leukemia or myelodysplastic syndromes. 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. 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: M D Anderson Cancer Center, Houston, Texas

  • Alpha-Beta Depleted T-cells and Cyclophosphamide following Stem Cell Transplant in Treating Patients with Hematological Malignancies

    This phase I / II trial studies alpha-beta depleted T-cells and cyclophosphamide following stem cell transplant in treating patients with hematological malignancies. Giving chemotherapy and total-body irradiation before a donor hematopoietic tem 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. The donated stem cells may also replace the patient’s immune cells and help destroy any remaining cancer cells. 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.
    Location: University of Alabama at Birmingham Cancer Center, Birmingham, Alabama

  • Donor Natural Killer Cell Infusion after Autologous CD133+ Selected Stem Cell Transplant in Treating Younger Patients with High Risk Solid Tumors or Lymphomas

    This pilot clinical trial studies whether a donor natural killer cell infusion can be safely used after autologous cluster of differentiation (CD)133+ selected stem cell transplant in treating younger patients with solid tumors or lymphomas that are likely to come back or spread. Giving chemotherapy before a stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. An autologous transplant means that stem cells are collected from the patient's blood and stored. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy. Stem cell selection is used to separate stem cells from other cells collected, which may include tumor cells. A natural killer cell is a type of white blood cell that has small particles with enzymes that can kill tumor cells. Adding a haploidentical donor (partially matched family member donor) natural killer cell infusion after an autologous stem cell transplant may help treat younger patients with high risk solid tumors or lymphomas.
    Location: St. Jude Children's Research Hospital, Memphis, Tennessee

  • Targeted Marrow Irradiation, Fludarabine Phosphate, and Busulfan before Donor Progenitor Cell Transplant in Treating Patients with Hematologic Malignancies

    This phase I trial studies the side effects and best dose of targeted marrow irradiation when given with fludarabine phosphate and busulfan before a donor progenitor cell transplant in treating patients with hematologic malignancies. Targeted marrow irradiation is a type of specialized radiation therapy that delivers a high dose of radiation directly to the cancer cells, which may kill more cancer cells and cause less damage to normal cells. Giving targeted marrow irradiation and chemotherapy drugs, such as fludarabine phosphate and busulfan, before a donor progenitor cell transplant may help stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's progenitor cells. When the healthy progenitor cells from a donor are infused into the patient they may help the patient's bone marrow make progenitor cells, red blood cells, white blood cells, and platelets.
    Location: Case Comprehensive Cancer Center, Cleveland, Ohio

  • Fludarabine Phosphate, Clofarabine, and Busulfan with Vorinostat in Treating Patients with Acute Leukemia in Remission or Relapse Undergoing Donor Stem Cell Transplant

    This phase I trial studies the side effects and best dose of vorinostat when given together with fludarabine phosphate, clofarabine, and busulfan in treating patients with acute leukemia that is under control (remission) or has returned (relapse) undergoing donor stem cell transplant. Vorinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as fludarabine phosphate, clofarabine, and busulfan, 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. Giving vorinostat together with fludarabine phosphate, clofarabine, and busulfan before a donor stem cell transplant may be a better treatment for patients with acute leukemia.
    Location: M D Anderson Cancer Center, Houston, Texas

  • Allogeneic or Haploidentical Stem Cell Transplant Followed by High-Dose Cyclophosphamide in Treating Patients with Relapsed or Refractory Acute Myeloid Leukemia

    This pilot phase I trial studies overall health status and how disease responds to a stem cell transplant when followed with a couple of doses of a drug called cyclophosphamide in patients with acute myeloid leukemia that has returned after all signs and symptoms of cancer have disappeared, although cancer may still be in the body (complete remission) or that has never fully responded to therapy. An allogeneic stem cell transplant is a procedure in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a genetically similar, but not identical, donor. This is often a sister or brother, but could be an unrelated donor. When a patient cannot find a donor who exactly matches their tissue type, half-matched related (haploidentical) donors, who are first degree relatives such as siblings, children, or parents, may be used. People who undergo a stem cell transplant can experience complications such as rejection of the stem cell transplant or severe graft-versus host disease (GVHD). Giving cyclophosphamide after haploidentical stem cell transplant may improve the outcomes of the transplant.
    Location: Siteman Cancer Center at Washington University, Saint Louis, Missouri

  • Partially-Matched Donor Stem Cell Transplant in Treating Younger Patients with High-Risk Hematologic Malignancies

    This pilot clinical trial studies partially-matched donor stem cell transplant in treating younger patients with high-risk hematologic malignancies. Giving chemotherapy and / or 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving cyclophosphamide, tacrolimus, and mycophenolate mofetil after the transplant may stop this from happening.
    Location: Lurie Children's Hospital-Chicago, Chicago, Illinois


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