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 26-50 of 225

  • Study to Evaluate the Safety and Efficacy of the Coadministration of Ibrexafungerp (SCY-078) With Voriconazole in Patients With Invasive Pulmonary Aspergillosis

    Study to evaluate the safety and efficacy of coadminstration of SCY-078 with a mold-active azole (voriconazole) compared to voriconazole in patients with invasive pulmonary aspergillosis.
    Location: 4 locations

  • A Phase 1b / 2 Study of Alvocidib Plus Decitabine or Azacitidine in Patients With MDS

    Alvocidib, a cyclin-dependent kinase 9 (CDK 9) inhibitor, in time-sequential therapy demonstrated significant clinical activity in secondary AML patients with prior MDS. Patients with IPSS-R intermediate and above MDS have an increased risk of developing AML and may be treated with the same chemotherapy regimens used in patients with AML. Eight Phase I or II clinical trials have been completed in patients with AML, totaling more than 400 patients with both relapsed / refractory or newly diagnosed AML. Preclinical studies have demonstrated that decitabine exposure increased the expression of NOXA, which is a specific antagonist of the survival factor MCL 1. Pharmacologic downregulation of MCL-1 via CDK 9 inhibition, as well as upregulation of the MCL-1 antagonist, NOXA, following decitabine exposure may result in enhanced antileukemic activity in MCL-1-dependent malignancies.
    Location: 7 locations

  • Talazoparib for the Treatment of Cohesin-Mutated Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

    This phase I trial studies how well talazoparib works in treating patients with cohesion-mutated acute myeloid leukemia that has come back (relapsed) or does not respond to treatment (refractory) or myelodysplastic syndrome. Talazoparib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: 4 locations

  • Enasidenib in Treating Participants with IDH2-Mutant Myeloid Cancers after Stem Cell Transplant

    This phase I trials studies the side effects and best dose of enasidenib in treating participants with IDH2-mutant myeloid cancers after stem cell transplant. Enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: 4 locations

  • Azacitidine and Enasidenib in Treating Patients with IDH2-Mutant Myelodysplastic Syndrome

    This phase II trial studies the side effects and how well azacitidine and enasidenib work in treating patients with IDH2-mutant myelodysplastic syndrome. Azacitidine and enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: 4 locations

  • Efficacy and Safety of FG-4592 for Treatment of Anemia in Patients With Lower Risk MDS With Low Red Blood Cell Transfusion Burden

    The purpose of this study is to determine whether FG-4592 is safe and effective in the treatment of anemia in patients with Lower Risk Myelodysplastic Syndrome and Low Red Blood Cell Transfusion Burden.
    Location: 5 locations

  • Tagraxofusp-erzs, Azacitidine and Venetoclax for the Treatment of Untreated, Relapsed, or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

    This phase I trial studies the side effects and best dose of tagraxofusp-erzs (SL-401) when given together with azacitidine, or azacitidine and venetoclax, in treating patients with acute myeloid leukemia that is untreated, has come back (relapsed), or does not respond to treatment (refractory) or myelodysplastic syndrome. Combinations of biological substances in tagraxofusp-erzs may be able to carry cancer-killing substances directly to cancer cells. Drugs used in chemotherapy, such as 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. Venetoclax may stop the growth of cancer cells by blocking a protein called Bcl-2 needed for cell growth. Giving tagraxofusp-erzs with azacitidine and venetoclax may work better in treating patients with acute myeloid leukemia or myelodysplastic syndrome compared to standard therapy.
    Location: 4 locations

  • Clinical Trial of BP1001 in Combination With With Venetoclax Plus Decitabine in AML

    The primary objectives of this study are to assess: (1) whether the combination of BP1001 plus venetoclax plus decitabine provides greater efficacy (Complete Remission [CR], Complete Remission with incomplete hematologic recovery [CRi], Complete Remission with partial hematologic recovery [CRh], than venetoclax plus decitabine alone (by historical comparison) in participants with untreated AML that cannot or elect not to be treated with more intensive chemotherapy; (2) whether BP1001-based treatment provides greater efficacy (CR, CRi, CRh) than intensive chemotherapy (by historical comparison) in participants with refractory / relapsed AML.
    Location: 4 locations

  • Antigen-specific T Cell Therapy for AML or MDS Patients With Relapsed Disease After Allo-HCT

    This Research study is being done to characterize the safety, tolerability, and preliminary antitumor activity of the NEXI-001 T cell product (a new experimental therapy), which contains populations of CD8+ T cells targeting multiple leukemia associated antigen peptides in patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) who have relapsed disease after an allogeneic hematopoietic cell transplant (HCT). The study will enroll AML or MDS patients who have either Minimal Residual Disease (MRD) or relapsed disease after a human leukocyte antigen (HLA)-matched allogeneic HCT. Patients who have had an HLA-mismatched or haploidentical allogeneic HCT will not be eligible to participate in this study. Eligible patients for this study must also have ≥ 50% T-cell chimerism from the original donor at the time study entry. The enrolled patients will undergo bridging therapy for the purposes of disease control while the NEXI-001 T cell product is being manufactured. Choice of bridging therapy administered will be per the Investigator's discretion, but is limited to acceptable agents as specified in the protocol. Bridging therapy will be administered prior to lymphodepleting (LD) therapy, with the last dose of the bridging therapy administered ≥ 14 days prior to initiation of LD therapy. Within 72 hours after completing LD therapy, patients will receive a single IV infusion of the NEXI-001 T cell product.
    Location: 3 locations

  • Liposome-Encapsulated Daunorubicin-Cytarabine in Treating Participants with Higher Risk Myelodysplastic Syndrome Who Are Transplant Eligible

    This phase I trial studies the side effects of liposome-encapsulated daunorubicin-cytarabine in treating participants with higher risk myelodysplastic syndrome who are transplant eligible. Drugs used in chemotherapy, such as liposome-encapsulated daunorubicin-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.
    Location: 3 locations

  • A Trial to Evaluate the Potential Impact of Renal Impairment on the Pharmacokinetics and Safety of CPX-351

    This study evaluates the pharmacokinetics and safety of CPX-351 in patients with moderate or severe renal impairment.
    Location: 5 locations

  • Study of PDR001 and / or MBG453 in Combination With Decitabine in Patients With AML or High Risk MDS

    To characterize the safety and tolerability of 1) MBG453 as a single agent or in combination with PDR001 or 2) PDR001 and / or MBG453 in combination with decitabine in AML and high risk MDS patients, and to identify recommended doses for future studies.
    Location: 3 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

  • 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: 4 locations

  • A Study of PRT543 in Participants With Advanced Solid Tumors and Hematologic Malignancies

    This is a Phase 1 cohort, dose-escalation, dose-expansion study of PRT543 in patients with advanced cancers who have exhausted available treatment options. The purpose of this study is to define a safe dose and schedule to be used in subsequent development of PRT543.
    Location: 5 locations

  • Efficacy and Safety Study of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in ESA Naïve Subjects Who Require Red Blood Cell Transfusions

    The study will be conducted in compliance with the International Council for Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use / Good Clinical Practice (GCP) and applicable regulatory requirements. This is an interventional active-controlled, open-label, randomized Phase 3 study to compare the efficacy and safety of luspatercept (ACE-536) versus epoetin alfa for the treatment of anemia due to IPSS-R very low, low or intermediate risk MDS in ESA naïve subjects who require RBC transfusions. The study is divided into the Screening Period, a Treatment Period and a Post-Treatment Follow-up Period.
    Location: 3 locations

  • Ivosidenib as Maintenance Therapy in Treating Patients with IDH1-mutant Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia following Stem Cell Transplant

    This phase I trial studies the best dose and side effects of ivosidenib as maintenance therapy in treating patients with IDH1-mutant acute myeloid leukemia, myelodysplastic syndrome, or chronic myelomonocytic leukemia following stem cell transplant. Ivosidenib is an inhibitor of the protein IDH1. IDH1 is an enzyme that, when mutated, can overproduce metabolites (substances that help with metabolism) and compounds that contribute to the growth of cancer cells. Ivosidenib may help block the over production of these substances and possibly reduce the chances of relapse in patients with IDH1-mutant myeloid cancers.
    Location: 4 locations

  • Trial of AB-110 in Adults With Hematologic Malignancies Undergoing Cord Blood Transplantation

    A phase 1b, open label, multi-center trial of AB-110 in adults with hematologic malignancies, including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplasia (MDS) undergoing cord blood transplantation. Subjects will receive unmanipulated cord blood (UCB) and AB-110 expanded CD34 enriched hematopoietic progenitor cells (HSPC).
    Location: 3 locations

  • Dose-escalation Study of Oral Administration of LP-108 in Patients With Relapsed or Refractory Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML)

    A Phase 1, Multi-center, Open-label, Dose Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Clinical Activity of Orally Administered LP-108 in Subjects with Relapsed or Refractory Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML)
    Location: 2 locations

  • Cell Therapy (CIML NK Cells) for the Treatment of Recurrent Myeloid Disease after Donor Blood Stem Cell Transplant

    This phase I trial studies the side effects and best dose of cell therapy (CIML NK cells) in treating patients with myeloid disease that has come back (recurrent) after undergoing a donor blood stem cell transplant. Drugs used in chemotherapy, such as fludarabine 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. CIML NK cells may recognize and kill cancer cells. Aldesleukin may stimulate white blood cells, including natural killer cells, to kill myeloid cells. Giving CIML NK cells with aldesleukin may increase the levels of NK cells and kill more myeloid cells.
    Location: 2 locations

  • T Cell-Depleted Donor Lymphocyte Infusion and Ipilimumab in Treating Patients with Myeloid Disease Relapse after Donor Stem Cell Transplant

    This phase I trial studies the side effects and best dose of T cell-depleted donor lymphocyte infusion and ipilimumab in treating patients with acute myeloid leukemia, myelodysplastic syndrome, myeloproliferative neoplasm, chronic myelomonocytic leukemia, or myelofibrosis that has come back after a donor stem cell transplant. Previously, patients who have relapsed after a donor stem cell transplant have been given infusions of donor white blood cells called donor lymphocyte infusions (DLI) as a way to boost their donor’s immune function and fight the cancer. This immune function can be suppressed by natural anti-inflammatory immune cells (T cells) that are present in the DLI product. Depleting the number of T cells in the DLI product may work better in fighting the cancer. Immunotherapy with monoclonal antibodies, such as ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. This trial determines the highest dose of ipilimumab that can be given safely in several courses and whether ipilimumab may help the donor white blood cells kill the cancer cells.
    Location: 2 locations

  • Pevonedistat and Belinostat in Treating Patients with Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

    This phase I trial studies side effects and best dose of pevonedistat and belinostat in treating patients with acute myeloid leukemia or myelodysplastic syndrome that has come back or does not respond to treatment. Drugs used in chemotherapy, such as pevonedistat and belinostat, 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: 2 locations

  • Venetoclax, Busulfan, Fludarabine, and Azacitidine in Treating Patients with High Risk Acute Myeloid Leukemia, Myelodysplastic Syndrome, and Myelodysplastic / Myeloproliferative Neoplasm Overlap Syndromes Undergoing Donor Stem Cell Transplantation

    This phase I trial studies the best dose and side effects of venetoclax when given together with busulfan, fludarabine, and azacitidine in treating patients with high risk acute myeloid leukemia, myelodysplastic syndrome, chronic myelomonocytic leukemia, or myelodysplastic syndrome / myeloproliferative neoplasm undergoing donor hematopoietic stem cell transplantation. Drugs used in chemotherapy, such as venetoclax, busulfan, fludarabine, and azacitidine 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.
    Location: 2 locations

  • Reduced Intensity Chemotherapy and Total Body Irradiation before TCR-alpha / beta+ T-lymphocytes Donor Transplant in Treating Participants with High-Risk Myeloid Diseases

    This phase I trial studies how well reduced intensity chemotherapy and total-body irradiation before allogeneic TCR alpha / beta-positive T-lymphocyte-depleted peripheral blood stem cells (TCR-alpha / beta+ T-lymphocytes donor transplant) works in treating participants with high-risk myeloid diseases. Giving chemotherapy such as anti-thymocyte globulin and fludarabine phosphate, as well as total-body irradiation 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 participant's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the participant they may help the participant'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). Removing the T cells from the donor cells before the transplant may stop this from happening.
    Location: 2 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