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 85
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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 or does not respond to treatment. Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
    Location: 46 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: 11 locations

  • SL-401 in Advanced, High Risk Myeloproliferative Neoplasms (Systemic Mastocytosis, Advanced Symptomatic Primary Eosinophilic Disorder, Myelofibrosis, Chronic Myelomonocytic Leukemia)

    This is a non-randomized open label multi-center study. Patients with high-risk myeloproliferative neoplasms (systemic mastocytosis [SM], advanced symptomatic primary eosinophilic disorder [PED], myelofibrosis [MF], and chronic myelomonocytic leukemia [CMML]) will be treated with SL-401, which will be administered as a brief intravenous infusion for 3 consecutive days initially every 21 days for 4 cycles; every 28 days for cycles 5-7; then every 42 days. Stage 1 will consist of a period in which several doses of SL-401 are evaluated. The Stage 2 portion will enroll up to 30 patients each with one of the two myeloproliferative malignancies: MF and CMML. In entirety, the Stage 2 portion will consist of up to 60 patients who will be treated at a maximum tolerated dose or maximum tested dose in which multiple dose-limiting toxicities are not observed (identified in Stage 1).
    Location: 9 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 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.
    Location: 7 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: 7 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: 9 locations

  • PRIMA-1 Analog APR-246 and Azacitidine in Treating Patients with TP53 Mutant Myeloid Cancers

    This phase Ib / II trial studies the side effects and best dose of PRIMA-1 analog APR-246 when given together with azacitidine and to see how well they work in treating patients with TP53 mutant myeloid cancers. Giving PRIMA-1 analog APR-246 and azacitidine may work better in treating patients with TP53 mutant myeloid cancers.
    Location: 6 locations

  • Low Dose Decitabine, Low Dose Azacitidine, or Standard Dose Azacitidine in Treating Patients with Transfusion-Dependent Myelodysplastic Syndrome or Best Supportive Care in Patients with Transfusion-Independent Myelodysplastic Syndrome

    This randomized phase II trial studies how well low dose decitabine, low dose azacitidine, or standard dose azacitidine works in treating patients with myelodysplastic syndrome (MDS) who need blood transfusion (transfusion-dependent) compared to best supportive care in patients with MDS who do not need blood transfusion (transfusion-independent). Drugs used in chemotherapy, such as 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. It is not yet known whether low dose decitabine, low dose azacitidine, or standard dose azacitidine is most effective in treating or offering best supportive care for patients with myelodysplastic syndrome.
    Location: 6 locations

  • Ruxolitinib Phosphate in Treating Patients with Chronic Neutrophilic Leukemia or Atypical Chronic Myeloid Leukemia

    This phase II trial studies how well ruxolitinib phosphate works in treating patients with chronic neutrophilic leukemia (CNL) or atypical chronic myeloid leukemia (aCML). Ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cells to reproduce. This trial also studies the genetic makeup of patients. Certain genes in cancer cells may determine how the cancer grows or spreads and how it may respond to different drugs. Studying how the genes associated with CNL and aCML respond to the study drug may help doctors learn more about CNL and aCML and improve the treatment for these diseases.
    Location: 6 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

  • Umbralisib and Ruxolitinib Phosphate in Treating Patients with Primary Myelofibrosis, Post-Polycythemia Vera MF, Post-Essential Thrombocythemia MF, Myelodysplastic / Myeloproliferative Neoplasm, or Polycythemia Vera

    This phase I trial studies the side effects and best dose of umbralisib and ruxolitinib phosphate when given together in treating patients with primary myelofibrosis, post-polycythemia vera myelofibrosis (MF), post-essential thrombocythemia MF, myelodysplastic / myeloproliferative neoplasm or polycythemia vera. Umbralisib and ruxolitinib phosphate 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

  • 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

  • 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: 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

  • 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

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

    This randomized 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, 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: 5 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

  • A Phase II Trial of CD24Fc for the Prevention of Acute GVHD Following Myeloablative Allogeneic HSCT

    This is a multicenter prospective randomized phase IIa / II Expansion clinical trial designed to determine the MTD (Maximum Tolerated Dose) of CD24Fc for acute GVHD (Graft Versus Host Disease) prophylaxis. Funding Source - FDA OOPD
    Location: 4 locations

  • Study of Lenzilumab in Previously Treated Patients With Chronic Myelomonocytic Leukemia (CMML)

    This is a multicenter, open-label, repeat-dose, Phase 1 Dose Escalation Study to evaluate safety, pharmacokinetics, and clinical activity.
    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

  • CPX-351 in Treating Patients with Relapsed or Refractory High Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

    This phase I trial studies best dose and side effects of liposome-encapsulated daunorubicin-cytarabine (CPX-351) and how well it works in treating patients with high risk myelodysplastic syndrome or chronic myelomonocytic leukemia that has come back or has not responded to treatment. 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: M D Anderson Cancer Center, Houston, Texas

  • A Study of LB-100 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)

    The purpose of this study is to test the safety and efficacy (benefits) of an investigational drug LB-100, for treatment of myelodysplastic syndromes. LB-100 has previously been administered to patients with various solid tumors. In this study, LB-100 will be administered as an intravenous infusion over 120 minutes. This study will be conducted in 2 phases. In phase Ib, escalating doses of LB-100 will be administered to patients to study the safety and to determine a safe dose of LB-100. In phase 2, patients will be administered LB-100 at the dose that was found to be safe in phase Ib. The efficacy (benefits) and safety of LB-100 will be determined in this phase of the study.
    Location: Moffitt Cancer Center, Tampa, Florida


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