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 82
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  • 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: 12 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: 8 locations

  • Study to Investigate the Safety and Clinical Activity of GSK3326595 and Other Agents to Treat Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukaemia (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 subjects with cancer. Myelodysplastic syndrome and acute myeloid leukaemia are bone marrow neoplasms for which novel, effective therapies are desperately needed. This is an open-label, multicentre, multi-part study to evaluate the safety, tolerability, and clinical activity of GSK3326595 in subjects with relapsed and refractory MDS, chronic myelomonocytic leukaemia (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 subjects with AML whose disease contains mutations in spliceosome proteins.
    Location: 6 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: 7 locations

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

    This phase Ib / II 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 have fails primary therapy and that does not respond to treatment or has come back. Pevonedistat and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: 5 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

  • Study of ASTX727 vs IV Decitabine in MDS, CMML, and AML

    Multicenter, randomized, open-label, crossover PK study of ASTX727 versus IV decitabine. Adult subjects who are candidates to receive IV decitabine will be randomized 1:1 to receive the ASTX727 tablet Daily×5 in Cycle 1 followed by IV decitabine 20 mg / m^2 Daily×5 in Cycle 2, or the converse order. After completion of PK studies during the first 2 treatment cycles, subjects will continue to receive treatment with ASTX727 from Cycle 3 onward (in 28-day cycles) until disease progression, unacceptable toxicity, or the subject discontinues treatment or withdraws from the study.
    Location: 5 locations

  • Guadecitabine and Atezolizumab in Treating Patients with Advanced Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia That Is Refractory or Relapsed

    This phase I / II trial studies the side effects and best dose of guadecitabine when given together with atezolizumab and to see how well they work in treating patients with myelodysplastic syndrome or chronic myelomonocytic leukemia that has spread to other places in the body and has come back or does not respond to treatment. Guadecitabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Monoclonal antibodies, such as atezolizumab, may interfere with the ability of cancer cells to grow and spread. Giving guadecitabine and atezolizumab may work better in treating patients with myelodysplastic syndrome or chronic myelomonocytic leukemia.
    Location: 5 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

  • 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

  • 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: 3 locations

  • Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML), or Low-Blast Acute Myelogenous Leukemia (AML)

    The purpose of this study is to determine whether the combination of pevonedistat and azacitidine improves event-free survival (EFS) when compared with single-agent azacitidine (An event is defined as death or transformation to AML in participants with MDS or CMML, whichever occurs first, and is defined as death in participants with low-blast AML).
    Location: 3 locations

  • Tipifarnib in Subjects With Chronic Myelomonocytic Leukemia, Other MDS / MPN, and Acute Myeloid Leukemia

    A Phase 2 study to investigate the antitumor activity in terms of overall response rate (ORR) of tipifarnib in approximately 36 eligible subjects with Myelodysplastic / Myeloproliferative Neoplasias (MDS / MPN), including Chronic Myelomonocytic Leukemia (CMML), and 36 eligible subjects with Acute Myeloid Leukemia (AML). Subjects (amendment 3 Cohorts 1-4) will receive tipifarnib administered at a dose of 400 mg, orally with food, twice a day (bid) for 21 days in 28 day cycles.
    Location: 5 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 and Fludarabine in Treating Patients with 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 and fludarabine in treating patients with 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 and fludarabine, 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

  • Pembrolizumab in Treating Patients with Advanced Myeloproliferative Tumors

    This phase II trial studies the side effects of pembrolizumab and how well it works in treating patients with myeloproliferative tumors that spread to other places in the body. Monoclonal antibodies, such as pembrolizumab, may interfere with the ability of tumor cells to grow and spread.
    Location: 2 locations

  • Lenalidomide and Eltrombopag Olamine in Treating Patients with Symptomatic Anemia in Low or Intermediate Myelodysplastic Syndrome

    This phase II trial studies how well lenalidomide and eltrombopag olamine works in treating patients with symptomatic anemia in low or intermediate myelodysplastic syndrome. Lenalidomide may stimulate the immune system in different ways and stop cancer cells from growing. Eltrombopag olamine may increase the number of white blood cells and platelets found in bone marrow or peripheral blood. Giving lenalidomide and eltrombopag olamine may be an effective treatment for myelodysplastic syndrome.
    Location: 3 locations

  • 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

  • Venetoclax and Azacitidine for the Treatment of High-Risk Recurrent or Refractory Myelodysplastic Syndrome

    This phase I / II trial studies the side effects and best dose of venetoclax when given together with azacitidine in treating patients with high-risk myelodysplastic syndrome that has come back (recurrent) or does not respond to treatment (refractory). Drugs used in chemotherapy, such as venetoclax 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: M D Anderson Cancer Center, Houston, Texas

  • Giving Chemotherapy and rATG for a Shortened Amount of Time before a Donor Stem Cell Transplantation for the Treatment of Patients with Blood Cancers

    This phase I trial studies the side effects of giving chemotherapy and a drug called rATG for a shorter period of time before a donor stem cell transplant in treating patients with blood cancers. This study will also look at whether the condensed regimen can shorten hospitalization following the transplantation. A chemotherapy regimen with the drugs busulfan, melphalan, and fludarabine may kill cancer cells in the body, making room in the bone marrow for new blood stem cells to grow and reducing the chance of transplanted cell rejection. The chemotherapy drugs work to interrupt the DNA (genetic information) in the cancer cells, stopping the cells from dividing and causing them to die. rATG targets and deactivates white blood cells called T cells that survive the chemotherapy. T cells may see the donor’s cells as foreign, causing a serious condition called graft-versus-host disease (GVHD). rATG helps prevent the donor stem cells from being rejected. Giving chemotherapy and rATG for a shorter period of time before a donor stem cell transplantation may help in reducing the number of side effects and shortening hospitalization following the transplantation.
    Location: Memorial Sloan Kettering Cancer Center, New York, New York

  • 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: 3 locations

  • PLX51107 and Azacitidine in Treating Patients with Acute Myeloid Leukemia or Myelodysplastic Syndrome

    This phase I trial studies the side effects and best dose of PLX51107 and how well it works with azacitidine in treating patients with acute myeloid leukemia or myelodysplastic syndrome. PLX51107 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. 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. Giving PLX51107 and azacitidine may work better than azacitidine alone in treating patients with acute myeloid leukemia or myelodysplastic syndrome.
    Location: M D Anderson Cancer Center, Houston, Texas


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