Treatment Clinical Trials for Myelodysplastic Syndrome

Clinical trials are research studies that involve people. The clinical trials on this list are for myelodysplastic syndrome 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 176-200 of 201

  • Combination Chemotherapy and Total Body Irradiation in Treating Patients with Fanconi Anemia Undergoing Donor Stem Cell Transplant

    This phase II / III trial studies how well combination chemotherapy and total body irradiation works in treating patients with Fanconi anemia undergoing donor stem cell transplant. Drugs used in the chemotherapy, such as cyclophosphamide and fludarabine phosphate, 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 and total-body irradiation before a donor 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.
    Location: University of Minnesota / Masonic Cancer Center, Minneapolis, Minnesota

  • A Safety Study of SEA-CD70 in Patients With Myeloid Malignancies

    This trial will look at a drug called SEA-CD70 to find out if it is safe for patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). It will study SEA-CD70 to find out what its side effects are and if it works for AML and MDS. A side effect is anything the drug does besides treating cancer. This study will have three groups or "parts." Part A will find out how much SEA-CD70 should be given to patients. Part B will use the dose found in Part A to find out how safe SEA-CD70 is and if it works to treat patients with MDS. Part C will use the dose found in Part A to find out how safe SEA-CD70 is and if it works to treat patients with AML.
    Location: 2 locations

  • Azacitidine Plus Nivolumab Following Reduced-intensity Allogeneic PBSC Transplantation for Patients With AML and High-risk Myelodysplasia

    This is a phase I clinical trial that will define the maximum tolerated dose (MTD) and investigate the feasibility and safety of the combination of nivolumab and azacitidine after reduced-intensity allogeneic PBSC transplantation. Dose escalation will follow a traditional 3+3 design. The investigators will first escalate the dose of single agent nivolumab to determine its MTD (if any, at the doses tested), with an expanded cohort at the MTD or highest dose tested. The investigators will then combine escalating azacitidine in combination of with nivolumab at its determined MTD or highest dose tested in earlier cohorts, and expand the highest dose cohort tested with the combination. Patients will be treated according to the dose level cohorts described in the protocol.
    Location: 2 locations

  • First in Human Testing of Dose-escalation of SAR440234 in Patients With Acute Myeloid Leukemia, Acute Lymphoid Leukemia and Myelodysplastic Syndrome

    Primary Objective: - Dose escalation: To determine the maximum tolerated dose (MTD) of SAR440234 administered as a single agent in patients with R / R AML (relapsed or refractory acute myeloid leukemia), HR-MDS (high risk myelodysplastic syndrome), or B-ALL (B-cell acute lymphoblastic leukemia), and determine the recommended phase 2 dose (RP2D) for the subsequent Expansion part. - Expansion part: To assess the activity of single agent SAR440234 at the RP2D in patients with R / R AML or HR-MDS. Secondary Objective: - To characterize the safety profile including cumulative adverse drug reactions. - To evaluate the potential immunogenicity of SAR440234. - To assess any preliminary evidence of hematologic response in the Dose Escalation Part.
    Location: M D Anderson Cancer Center, Houston, Texas

  • A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple- Dose BIVV009 in Participants With Chronic Immune Thrombocytopenia (ITP)

    The purpose of this study is to explore the safety, preliminary clinical benefit, and activity of BIVV009 in patients with chronic immune thrombocytopenia.
    Location: University of Pittsburgh Cancer Institute (UPCI), Pittsburgh, Pennsylvania

  • IGF-MTX Conjugate in the Treatment of Myelodysplastic Syndrome

    The primary objective of this study is to determine the safety and tolerability of utilizing the insulin-like growth factor-1-methotrexate conjugate, 765IGF-MTX for the treatment of advanced, previously treated myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML) and oligoblastic acute myelogenous leukemia (oligoblastic AML or O-AML), including determining the maximum tolerated dose (MTD).
    Location: Mayo Clinic, Rochester, Minnesota

  • Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome

    An open label single arm study to assess efficacy and safety of BL-8040 on top of standard immunotherapy regimen of hATG, cyclosporine and steroids in patients with Hypoplastic MDS and AA over the course of a six month (180 day) treatment period.
    Location: See Clinical Trials.gov

  • Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies

    The purpose of this study is to assess the safety, tolerability, and clinical activity of OMS721 in subjects with thrombotic microangiopathies (TMA), which include atypical hemolytic uremic syndrome (aHUS), thrombotic thrombocytopenia (TTP), and hematopoietic stem cell transplant -associated TMA (HSCT-associated TMA). The study will also evaluate clinical activity, pharmacokinetics (PK), pharmacodynamics (PD), and anti-drug antibody response (ADA).
    Location: See Clinical Trials.gov

  • Study of AR-67 (DB-67) in Myelodysplastic Syndrome (MDS)

    The purpose of this study is to determine if AR-67 is effective in the treatment for patients with MDS.
    Location: UCLA / Jonsson Comprehensive Cancer Center, Los Angeles, California

  • 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

  • An Open-Label, Multicenter, Extension Study for Subjects Who Participated in Prior Clinical Studies of ASTX727 (Standard Dose)

    Extension study for subjects who participated in a previous Astex-sponsored clinical study of ASTX727 (including, but not limited to ASTX727-01, ASTX727-02, ASTX727-04).
    Location: 4 locations

  • Rivogenlecleucel Donor Lymphocyte Immunotherapy in Treating Patients with Recurrent Blood Cancers after Stem Cell Transplant

    This phase I trial studies the side effects and best dose of rivogenlecleucel, and how well it works, in treating patients with blood cancer that has come back (recurrent) after stem cell transplant. Donor T-cell therapy (rivogenlecleucel) may help control transplant-related infections after stem cell transplant.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • A Study of Aezea® (Cenersen) in Transfusion Dependent Anemia Associated With Myelodysplastic Syndrome (MDS)

    The purpose of the study is to test the safety of six cycles of cenersen treatment and to begin to test the hypothesis that intermittent administration of cenersen may lead to a reduced dependence on transfusion.
    Location: See Clinical Trials.gov

  • Decitabine with Ruxolitinib or Fedratinib for the Treatment of Accelerated / Blast Phase Myeloproliferative Neoplasms

    This phase II trial studies how well decitabine with ruxolitinib or fedratinib works before hematopoietic stem cell transplant in treating patients with accelerated / blast phase myeloproliferative neoplasms (tumors). Drugs used in chemotherapy, such as decitabine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Ruxolitinib and fedratinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving chemotherapy before a donor hematopoietic 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. Decitabine, with ruxolitinib or fedratinib, may work better than multi-agent chemotherapy or no pre-transplant therapy, in treating patients with accelerated / blast phase myeloproliferative neoplasms.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • SNS-301 Monotherapy in High Risk MDS and CMML

    To evaluate safety, immunogenicity and anti-tumor responses of intradermally delivered SNS-301 in patients with ASPH+ high risk MDS and CMML.
    Location: UCLA / Jonsson Comprehensive Cancer Center, Los Angeles, California

  • Cord Blood Transplant with OTS for the Treatment of HIV Positive Hematologic Cancers

    This phase II trial studies the side effects of a cord blood transplant using OTS and to see how well it works in treating patients with human immunodeficiency virus (HIV) positive hematologic (blood) cancers. After a cord blood transplant, the immune cells, including white blood cells, can take a while to recover, putting the patient at increased risk of infection. OTS consists of blood stem cells that help to produce mature blood cells, including immune cells. Drugs used in chemotherapy, such as fludarabine, cyclophosphamide, and thiotepa, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Total body irradiation is a type of whole-body radiation. Giving chemotherapy and total-body irradiation before a cord blood transplant with OTS may help to kill any cancer cells that are in the body and make room in the patient’s bone marrow for new stem cells to grow and reduce the risk of infection.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • Donor Natural Killer Cell Therapy in Treating Patients with Recurrent or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

    This phase I trial studies the best dose of donor natural killer (NK) cell therapy and to see how well it works in treating patients with acute myeloid leukemia that has come back (recurrent) or has not responded to treatment (refractory), or myelodysplastic syndrome. Natural killer cells are a type of immune cell. Immunotherapy with genetically modified NK cells from donors may induce changes in the body’s immune system and may interfere with the ability of cancer cells to grow and spread.
    Location: Ohio State University Comprehensive Cancer Center, Columbus, Ohio

  • Salsalate, Venetoclax, and Decitabine or Azacitidine for the Treatment of Acute Myeloid Leukemia or Advanced Myelodysplasia / Myeloproliferative Disease

    This phase II trial studies the side effects of salsalate when added to venetoclax and decitabine or azacitidine in treating patients with acute myeloid leukemia or myelodysplasia / myeloproliferative disease that has spread to other places in the body (advanced). Drugs used in chemotherapy, such as salsalate, venetoclax, decitabine, and azacitidine 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: Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey

  • Liposomal Cytarabine and Daunorubicin (CPX-351) and Quizartinib for the Treatment of Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome

    This phase I / II trial studies the side effects and best dose of CPX-351 in combination with quizartinib for the treatment of acute myeloid leukemia and high risk myelodysplastic syndrome. CPX-351, composed of chemotherapy drugs daunorubicin and cytarabine, works 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. Quizartinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. The goal of this study is to learn if the combination of CPX-351 and quizartinib can help to control acute myeloid leukemia and myelodysplastic syndrome.
    Location: M D Anderson Cancer Center, Houston, Texas

  • Comparing ATG or Post-Transplant Cyclophosphamide to Calcineurin Inhibitor-Methotrexate as GVHD Prophylaxis after Myeloablative Unrelated Donor Peripheral Blood Stem Cell Transplantation

    This phase II trial studies how well 3 different drug combinations prevent graft versus host disease (GVHD) after donor stem cell transplant. Calcineurin inhibitors, such as cyclosporine and tacrolimus, may stop the activity of donor cells that can cause GVHD. Chemotherapy drugs, such as cyclophosphamide and methotrexate, may also stop the donor cells that can lead to GVHD while not affecting the cancer-fighting donor cells. Immunosuppressive therapy, such as anti-thymocyte globulin (ATG), is used to decrease the body's immune response and reduces the risk of GVHD. It is not yet known which combination of drugs: 1) ATG, methotrexate, and calcineurin inhibitor 2) cyclophosphamide and calcineurin inhibitor, or 3) methotrexate and calcineurin inhibitor may work best to prevent graft versus host disease and result in best overall outcome after donor stem cell transplant.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • Irradiated Donor Cells following Stem Cell Transplant in Controlling Cancer in Patients with Hematologic Malignancies

    This pilot clinical trial studies the side effects of irradiated donor cells following stem cell transplant in controlling cancer in patients with hematologic malignancies. Transfusion of irradiated donor cells (immune cells) from relatives may cause the patient's cancer to decrease in size and may help control cancer in patients receiving a stem cell transplant.
    Location: Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey

  • Ascorbic Acid in Treating Patients with Intermediate or High Risk Myelodysplastic Syndrome with TET2 Mutations

    This phase Ib / IIa trial studies the side effects of ascorbic acid and to see how well it works in treating patients with intermediate or high risk myelodysplastic syndrome with TET2 Mutations. Ascorbic acid is a natural water-soluble vitamin (vitamin C) that may help patients with myelodysplastic syndrome by lowering the activity of deoxyribonucleic acid (DNA) expression needed to make additional cells for cancer.
    Location: 2 locations

  • Azacitidine with or without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients with Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

    This randomized phase II / III trial studies how well azacitidine with or without nivolumab or midostaurin, or decitabine and cytarabine alone work in treating older patients with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome. Drugs used in chemotherapy, such as azacitidine, decitabine, and cytarabine, 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. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body’s immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Midostaurin may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving azacitidine with or without nivolumab or midostaurin, or decitabine and cytarabine alone may kill more cancer cells.
    Location: 490 locations

  • Three Dosing Schedules of Oral Rigosertib in MDS Patients

    This study will compare the dosing regimen of oral rigosertib, which has been used in other studies of lower risk Myelodysplastic Syndrome (MDS), with 2 new dosing regimens to determine if one of the new regimens gives improved results as measured by disease status, side effects, and analyses of blood and urine samples.
    Location: See Clinical Trials.gov

  • CPI-613 in Treating Patients with Myelodysplastic Syndrome Who Failed Previous Therapy

    This phase II pilot trial studies how well 6, 8-bis (benzylthio) octanoic acid (CPI-613) works in treating patients with myelodysplastic syndrome who failed previous therapy. Sometimes when chemotherapy or biological therapy is given, it does not stop the growth of tumor cells. The tumor is said to be resistant to treatment. 6, 8-bis (benzylthio) octanoic acid may interfere with the growth of cancer cells and may be an effective treatment for patients with myelodysplastic syndrome that did not respond to previous therapy.
    Location: Wake Forest University Health Sciences, Winston-Salem, North Carolina