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 107
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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 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: 13 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: 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: 12 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: 9 locations

  • Study of a Geriatric Assessment to Plan a Treatment Approach for Older People with Various Blood Disorders

    This phase II trial studies whether a geriatric assessment may be helpful in determining the best treatment approach in older patients (age 60+) with myelodysplastic syndromes, myeloproliferative neoplasms, or related blood disorders who are going to receive chemotherapy or another treatment to prepare the body for an allogeneic hematopoietic stem cell transplant (allo-HSCT). The geriatric assessment includes looking at patients’ cognitive function (thinking processes), physical function, mobility (ability to move the body), mood, nutrition, and current medications to help decide the type of treatment they’ll receive. Information from this study may help doctors better plan a treatment approach for older adults leading up to the stem cell transplant.
    Location: 7 locations

  • Study of IMGN632 in Patients With Untreated BPDCN and Relapsed / Refractory BPDCN

    This is an open-label, multi-center, Phase 1 / 2 study to determine the MTD and assess the safety, tolerability, PK, immunogenicity, and anti-leukemia activity of IMGN632 when administered as monotherapy to patients with CD123+ disease. The study is enrolling a pivotal cohort of frontline BPDCN patients and a cohort of relapsed / refractory BPDCN patients.
    Location: 7 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

  • 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: 5 locations

  • A Study of H3B-8800 in Participants With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia

    This is a Phase 1, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antitumor activity of H3B-8800 in subset of participants with Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML), or Chronic Myelomonocytic Leukemia (CMML). The study consists of two parts, a dose escalation part (Part 1) exploring a multiple once daily (QD) schedules and an expansion part (Part 2) exploring a twice daily (BID) schedule.
    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)

    This study will evaluate the safety, tolerability, and clinical activity of GSK3326595 in participants with relapsed and refractory MDS, chronic myelomonocytic leukemia (CMML), and AML. The study will be conducted in 2 parts: Part 1 will determine the clinical benefit rate (CBR) of GSK3326595 in monotherapy and Part 2 will be expanded to study GSK3326595 in combination with 5-Azacitidine which will be composed of a dose escalation phase followed by dose expansion cohort of GSK3326595.
    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

  • Study of Oral LY3410738 in Patients With Advanced Hematologic Malignancies With IDH1 or IDH2 Mutations

    This is an open-label, multi-center Phase 1 study of LY3410738, an oral, covalent IDH inhibitor, in patients with IDH1 and / or IDH2-mutant advanced hematologic malignancies who have received standard therapy
    Location: 9 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

  • 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

  • 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: 3 locations

  • A Study of TAS1553 in Subjects With Relapsed or Refractory Acute Myeloid Leukemia (AML) and Other Myeloid Neoplasms

    This is a Phase 1, 2-part, open-label, multicenter, first-in-human (FIH) study to assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of TAS1553 administered orally to participants ≥18 years of age with relapsed or refractory (R / R) acute myeloid leukemia (AML) or other myeloid neoplasms where approved therapies have failed or for whom known life-prolonging therapies are not available. The AML population includes de novo AML, secondary AML, and myelodysplastic syndrome (MDS)-transformed into AML. Other myeloid neoplasms include accelerated phase myeloproliferative neoplasms (MPN), and chronic or accelerated phase MPN-unclassifiable (MPN-U) and MDS-MPN. Blast crisis phase of MPNs are considered secondary AML and will be included in the AML cohort. Part 1 is a multicenter, sequential group treatment feasibility study with 1 treatment arm and no masking (dose escalation). Part 2 is a multicenter, two-stage, multiple group, dose confirmation study with 1 treatment arm and no masking (exploratory dose expansion).
    Location: 4 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

  • A Study of CFI-400945 With or Without Azacitidine or Decitabine in Patients With AML, MDS or CMML

    The purpose of this study is to test the safety of an investigational drug called CFI-400945 alone and in combination with azacitidine or decitabine
    Location: 3 locations

  • To Assess the Safety and Tolerability of INCB000928 in Participants With Myelodysplastic Syndromes or Multiple Myeloma.

    This Phase 1 / 2, open-label, dose-finding study is intended to evaluate the safety and tolerability, PK, PD, and efficacy of INCB000928 administered as monotherapy in participants with MDS or MM who are transfusion-dependent or present with symptomatic anemia.
    Location: 3 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

  • 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: 2 locations


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