Treatment Clinical Trials for Myelodysplastic/Myeloproliferative Disease

Clinical trials are research studies that involve people. The clinical trials on this list are for myelodysplastic/myeloproliferative disease 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 1-25 of 99
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  • Trametinib in Treating Patients with Relapsed or Refractory Juvenile Myelomonocytic Leukemia

    This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment (refractory). Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
    Location: 54 locations

  • A Phase 2 Study of CPI-0610 With and Without Ruxolitinib in Patients With Myelofibrosis

    Phase 1 Part (Complete): Open-label, sequential dose escalation study of CPI-0610 in patients with previously treated Acute Leukemia, Myelodysplastic Syndrome, Myelodysplastic / Myeloproliferative Neoplasms, and Myelofibrosis. Phase 2 Part: Open-label study of CPI-0610 with and without Ruxolitinib in patients with Myelofibrosis. CPI-0610 is a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins.
    Location: 10 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: 11 locations

  • Tagraxofusp (SL-401) in Patients With CMML or MF

    This multi-center, multi-arm trial is evaluating the safety and efficacy of tagraxofusp, a CD123-targeted therapy, in patients with either chronic myelomonocytic leukemia (CMML) or myelofibrosis (MF). There are two CMML cohorts, one enrolling patients with CMML (CMML-1 or CMML-2) who are refractory / resistant or intolerant to hypomethylating agents (HMA), hydroxyurea (HU), or intensive chemotherapy; and one enrolling treatment-naive patients with CMML (CMML-1 or CMML-2) with molecular features associated with poor prognosis. The MF cohort will enroll patients who are resistant / refractory or intolerant to approved JAK therapy (JAK1 / JAK2 or JAK2).
    Location: 11 locations

  • Study of IMGN632 in Patients With Relapse / Refractory AML, BPDCN, ALL, Other CD123+ Hem Malignancies

    This is an open-label, multi-center, Phase 1 / 2 study to determine the MTD and assess the safety, tolerability, PK, immunogenicity, and preliminary anti-leukemia activity of IMGN632 when administered as monotherapy to patients with CD123+ disease. The study is currently enrolling eligible AML, BPDCN and ALL patients.
    Location: 8 locations

  • A Safety, Tolerability and PK Study of DCC-2618 in Patients With Advanced Malignancies

    This is a Phase 1, open-label, first-in-human (FIH) dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary antitumor activity of DCC-2618, administered orally (PO), in adult patients with advanced malignancies. The study consists of 2 parts, a dose-escalation phase and an expansion phase.
    Location: 7 locations

  • Study to Investigate the Safety and Clinical Activity of GSK3326595 and Other Agents to Treat Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)

    GSK3326595 is a potent, selective, reversible inhibitor of the protein arginine methyltransferase 5 (PRMT5) / Methylosome protein 50 (MEP50) complex that is being tested as an oral treatment for human participants with cancer. Myelodysplastic syndrome and acute myeloid leukemia are bone marrow neoplasms for which novel, effective therapies are desperately needed. This is an open-label, multicenter, multi-part study to evaluate the safety, tolerability, and clinical activity of GSK3326595 in participants with relapsed and refractory MDS, chronic myelomonocytic leukemia (CMML), and hypoproliferative AML that has evolved from an antecedent MDS. The study will be conducted in two parts and at the end of Part 1, if pre-specified criteria are met, then the study will be expanded with three additional parts that will be opened in parallel (Part 2A, 2B and 2C). Part 1 is composed of a single-arm dose expansion cohort to determine the clinical benefit rate of GSK3326595. Part 2A is a randomized head-to-head Phase II evaluation of GSK3326595 compared to investigator's choice of best available care (BAC). Part 2B is composed of an abbreviated series of dose escalation cohorts followed by a single-arm dose expansion cohort to determine the overall response rate of the combination of GSK3326595 plus 5-azaciditine in newly-diagnosed MDS. Part 2C is a single-arm dose expansion study to evaluate the clinical activity of single-agent GSK3326595 in participants with AML whose disease contains mutations in spliceosome proteins.
    Location: 6 locations

  • A Study of ASTX030 (Cedazuridine in Combination With Azacitidine) in MDS, CMML, or AML

    Study ASTX030-01 is designed to move efficiently from Phase 1 to Phase 3. Phase 1 consists of an open-label Dose Escalation Stage (Stage A) using multiple cohorts at escalating dose levels of oral cedazuridine and azacitidine (only one study drug will be escalated at a time) followed by a Dose Expansion Stage (Stage B) of ASTX030. Phase 2 is a randomized open-label crossover study to compare oral ASTX030 to subcutaneous (SC) azacitidine. Phase 3 is a randomized open-label crossover study comparing the final oral ASTX030 tablet to SC azacitidine. The duration of the study is expected to be approximately 36 months.
    Location: 5 locations

  • AZD6738 for the Treatment of Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia Progressing on Standard Therapy

    This phase Ib trial studies the side effects, best dose, and response to AZD6738 in treating patients with myelodysplastic syndrome or chronic myelomonocytic leukemia progressing on standard therapy. DNA is the genetic material that serves as the body’s instruction book. Cancer is caused by changes (mutations) to genes (DNA) that control the way cells function. AZD6738 blocks a protein called ATR. ATR notices when there is injury to DNA and works to repair that damage. Studies done in a laboratory setting and cell lines suggest that myelodysplastic syndrome and chronic myelomonocytic leukemia cells rely specifically on the ATR pathway (a network of genes that interact with ATR) to fix DNA damage and survive; by inhibiting ATR with AZD6738, myelodysplastic syndrome or chronic myelomonocytic leukemia cells appear to selectively accumulate DNA damage and die, but healthy cells appear to be less sensitive to this drug. Inhibiting ATR may be a way to selectively target myelodysplastic syndrome or chronic myelomonocytic leukemia cells for treatment.
    Location: 5 locations

  • Pevonedistat and Azacitidine in Treating Patients with Refractory or Relapsed Myelodysplastic Syndrome or Myelodysplastic Syndrome / Myeloproliferative Neoplasm Who Fail Primary Therapy

    This phase II trial studies how well pevonedistat and azacitidine work in treating patients with myelodysplastic syndrome or myelodysplastic syndrome / myeloproliferative neoplasm that has failed primary therapy, that does not respond to treatment (refractory), or has come back (recurrent). Pevonedistat and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: 5 locations

  • Ruxolitinib in Treating Patients with Chronic Myelomonocytic Leukemia

    This phase II trial studies how well ruxolitinib works in treating patients with chronic myelomonocytic leukemia. Ruxolitinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: 4 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

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

    This phase I trials studies the side effects and best dose of enasidenib in treating patients 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

  • IO-202 as Monotherapy in Patients in AML and CMML

    To assess safety and tolerability at increasing dose levels of IO-202 in successive cohorts of participants with relapsed or refractory monocytic AML and CMML in order to estimate the maximum tolerated dose (MTD) or maximum administered dose (MAD) and select the recommended Phase 2 dose (RP2D) and dose schedule as monotherapy.
    Location: 6 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: 3 locations

  • Ruxolitinib, Decitabine, and Donor Lymphocyte Infusion in Treating Patients with Relapsed Acute Myeloid Leukemia or Myelodysplastic Syndrome after Stem Cell Transplant

    This phase II trial studies how well ruxolitinib, decitabine, and donor white blood cells (donor lymphocyte infusion [DLI]) work in treating patients with acute myeloid leukemia or myelodysplastic syndrome that has come back after a stem cell transplant. Patients who have relapsed after a stem cell transplant commonly receive an infusion of immune cells from the original donor called a DLI. A DLI uses high dose chemotherapy prior to the infusion which increases the risk of graft versus host disease, a condition in which the transplanted cells attack the recipient’s body. While the cancer responds temporarily to high dose chemotherapy alone, it hasn’t been shown to bring about long-term remission. Instead of high dose chemotherapy, this study pairs DLI with decitabine, another chemotherapy drug, and adds ruxolitinib. Ruxolitinib is a type of drug called a "JAK" inhibitor and may help prevent graft-versus host disease. Giving ruxolitinib with decitabine and a DLI may decrease the risk of graft-versus host disease and increase the chances of remission.
    Location: 4 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

  • 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

  • A Study to Investigate BAY2402234, a Dihydroorotate Dehydrogenase (DHODH) Inhibitor, in Myeloid Malignancies

    The primary objective is to determine the safety, tolerability, pharmacokinetics, maximum tolerated dose (MTD), or pharmacological active dose (PAD) of BAY2402234 in patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myelomonocytic leukemia (CMML). The secondary objective is to evaluate evidence of clinical efficacy associated with BAY2402234 in patients with AML (defined as Complete remission, Complete remission with partial hematologic recovery), and MDS (defined as hematological improvement).
    Location: 2 locations

  • Multi-institutional Prospective Research of Expanded Multi-antigen Specifically Oriented Lymphocytes for the Treatment of VEry High Risk Hematopoietic Malignancies

    This Phase I dose-escalation trial is designed to evaluate the safety of administering rapidly -generated tumor multi-antigen associated -specific cytotoxic T lymphocytes, to HSCT recipients (Arm A) or future HSCT recipients (Arm B) for the treatment of high-risk or relapsed or refractory hematopoietic malignancies. In addition to safety, this study will also evaluate if event-free survival (EFS) is improved with TAA-T administration at six months after HSCT for patients with high risk AML and MDS (Arm C).
    Location: 2 locations

  • Topotecan Hydrochloride and Carboplatin with or without Veliparib in Treating Advanced Myeloproliferative Disorders and Acute Myeloid Leukemia or Chronic Myelomonocytic Leukemia

    This phase II trial studies how well topotecan hydrochloride and carboplatin with or without veliparib work in treating patients with myeloproliferative disorders that have spread to other places in the body and usually cannot be cured or controlled with treatment (advanced), and acute myeloid leukemia or chronic myelomonocytic leukemia. Drugs used in chemotherapy, such as topotecan hydrochloride and carboplatin, 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. Veliparib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving topotecan hydrochloride, carboplatin, and veliparib may work better in treating patients with myeloproliferative disorders and acute myeloid leukemia or chronic myelomonocytic leukemia compared to topotecan hydrochloride and carboplatin alone.
    Location: 2 locations

  • Chemotherapy and Cord Blood Transplant in Children and Young Adults with Hematologic Malignancies or Non-malignant Diseases

    This phase II trial studies the effect of chemotherapy and a cord blood transplant in children and young adults with hematologic malignancies or non-malignant diseases. Chemotherapy drugs, such as clofarabine, fludarabine, 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. Before receiving stem cells, the standard process, called cytoreduction, is to receive high doses of chemotherapy. This helps to make room in the bone marrow for new blood stem cells to grow, helps prevent the body from rejecting the transplanted cells, and helps kill any cancer cells that are in the body. This is called a conditioning regimen. However, high doses of chemotherapy can have serious side effects. This study may help researchers learn whether combining the chemotherapy drugs clofarabine, fludarabine, and busulfan is a safe and effective way to reduce the side effects from receiving a conditioning regimen in children and young adults receiving cord blood transplants.
    Location: Memorial Sloan Kettering Cancer Center, New York, New York


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