Treatment Clinical Trials for Myelodysplastic/Myeloproliferative Disease

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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 70
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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: 30 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: 7 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

  • 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: 5 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: 5 locations

  • Ibrutinib and Azacitidine in Treating Patients with Higher Risk Myelodysplastic Syndrome Who Were Previously Treated or Untreated and Unfit for or Refused Intense Therapy

    This phase Ib trial studies the side effects and best dose of ibrutinib when given together with azacitidine in treating patients with myelodysplastic syndrome that is likely to occur or spread (higher risk) and who were previously treated or untreated and unfit for or refused intense therapy. Ibrutinib and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: 5 locations

  • A Combination Study of PF-04449913 (Glasdegib) and Azacitidine In Untreated MDS, AML and CMML Patients

    This multi center open label Phase 1b study is designed to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of glasdegib (PF-04449913) when combined with azacitidine in patients with previously untreated Higher Risk Myelodysplastic Syndrome (MDS), Acute Myeloid Leukemia (AML), or Chronic Myelomonocytic Leukemia (CMML). This clinical study includes two components: (a) a safety lead in cohort (LIC) and (b) an expansion phase with an AML cohort and an MDS cohort.
    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 Phase II Trial of CD24Fc for Prevention of Acute Graft-versus-Host Disease Following Myeloablative Allogeneic Hematopoietic Stem Cell Transplant

    This is a multicenter prospective randomized phase IIa / IIb clinical trial designed to determine the MTD (Maximum Tolerated Dose) of CD24Fc for acute GVHD (Graft Versus Host Disease) prophylaxis.
    Location: 3 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: 4 locations

  • Chemotherapy in Treating Patients with Myelodysplastic Syndrome before Donor Stem Cell Transplant

    This randomized clinical trial studies different chemotherapies in treating patients with myelodysplastic syndrome before donor stem cell transplant. Giving chemotherapy before a donor stem cell transplant helps stop the growth of cancer cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells, and may prevent the myelodysplastic syndrome from coming back after the transplant. 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.
    Location: 3 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

  • A Phase I, Two-part Study to Determine the Recommended Dose and Evaluate the Safety and Tolerability of a Novel Oral Arsenic Trioxide Formulation (ORH-2014) in Subjects With Advanced Hematological Disorders

    Part 1 will be conducted as an open-label, non-randomized, non-placebo-controlled dose escalation study using pre-specified doses. Subjects with the following advanced hematological disorders and no available therapies, and who satisfy all inclusion / exclusion criteria will be enrolled. The purpose is to identify the recommended dose of oral ORH-2014 in subjects with advanced hematological disorders. Part 2 will be an expansion phase conducted as a single-arm, open-label study to further evaluate the safety and tolerability of ORH-2014 at the maximum tolerated dose (MTD) or recommended dose determined from Part 1 in the fasted state. Subjects with the same disease types as in Part 1 will be enrolled. All subjects will receive oral ORH-2014, in the fasted state, at the recommended dose for an initial period of up to 12 weeks. The purpose is to evaluate the safety and tolerability of oral ORH-2014 in a population of subjects with advanced hematological disorders when administered at the recommended dose.
    Location: 2 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: 4 locations

  • Tipifarnib in Subjects With Chronic Myelomonocytic Leukemia

    A Phase 2 study to investigate the antitumor activity in terms of overall response rate (ORR) of tipifarnib in approximately 20 eligible subjects with CMML. Subjects will receive tipifarnib administered orally, twice a day (bid) for 7 days in alternating weeks (Days 1-7 and 15-21) in 28 day cycles. In the absence of unmanageable toxicities, subjects may continue to receive tipifarnib treatment until disease progression. If a complete response is observed, therapy with tipifarnib will be maintained for at least 6 months beyond the start of response.
    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

  • Study of FF-10501-01 in Patients With Relapsed or Refractory Hematological Malignancies

    A Phase 1 / 2a Dose Escalation Study of FF-10501-01 in Patients with Relapsed or Refractory Hematological Malignancies to determine the safety and tolerability. A total of 6 cohorts will be enrolled in Phase 1 to establish the MTD. A total of 20 subjects with MDS / CMML treated at the RP2D are planned, including MDS / CMML subjects treated at the RP2D in Phase 1.
    Location: 2 locations

  • T-Cell Infusion and Donor Stem Cell Transplant in Treating Patients with High-Risk Hematologic Cancer

    This phase I / II trial studies the side effects and best way to give T-cell infusion and donor stem cell transplant and to see how well it works in treating patients with high-risk hematologic cancer. Giving chemotherapy and total-body irradiation (TBI) before a donor stem cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When certain stem cells from a related donor, that do not exactly match the patient's blood, are infused into the patient they may help the patient'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. Removing the T cells from the donor cells before transplant may stop this from happening. Giving an infusion of the donor's T cells (donor lymphocyte infusion) may help the patient's immune system see any remaining cancer cells as not belonging in the patient's body and destroy them (called graft-versus-tumor effect).
    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

  • 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: M D Anderson Cancer Center, Houston, Texas

  • DEC-205 / NY-ESO-1 Fusion Protein CDX-1401, Poly ICLC, Decitabine, and Nivolumab in Treating Patients with Myelodysplastic Syndrome or Acute Myeloid Leukemia

    This phase I trial studies the side effects of DEC-205 / NY-ESO-1 fusion protein CDX-1401, poly ICLC, decitabine, and nivolumab in treating patients with myelodysplastic syndrome or acute myeloid leukemia. DEC-205 / NY-ESO-1 fusion protein CDX-1401 is a vaccine that may help the immune system specifically target and kill cancer cells. Poly ICLC may help stimulate the immune system in different ways and stop cancer cells from growing. Drugs used in chemotherapy, such as decitabine, 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. Monoclonal antibodies, such as nivolumab, may interfere with the ability of cancer cells to grow and spread. Giving DEC-205 / NY-ESO-1 fusion protein CDX-1401, poly ICLC, decitabine, and nivolumab may work better in treating patients with myelodysplastic syndrome or acute myeloid leukemia.
    Location: Roswell Park Cancer Institute, Buffalo, New York

  • Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients with Blood Cancer

    This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer. Drugs used in chemotherapy, such as fludarabine phosphate 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. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving chemotherapy and 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 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. The donated stem cells may also replace the patient’s immune cells and help destroy any remaining cancer cells.
    Location: Roswell Park Cancer Institute, Buffalo, New York

  • Comparison of Triple GVHD Prophylaxis Regimens for Nonmyeloablative or Reduced Intensity Conditioning Unrelated Mobilized Blood Cell Transplantation

    This randomized phase II trial includes a blood stem cell transplant from an unrelated donor to treat blood cancer. The treatment also includes chemotherapy drugs, but in lower doses than conventional (standard) stem cell transplants. The researchers will compare two different drug combinations used to reduce the risk of a common but serious complication called "graft versus host disease" (GVHD) following the transplant. Two drugs, cyclosporine (CSP) and sirolimus (SIR), will be combined with either mycophenolate mofetil (MMF) or post-transplant cyclophosphamide (PTCy). This part of the transplant procedure is the main research focus of the study.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • Combination Chemotherapy, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients with Hematopoietic Malignancies Including Those That Are Challenging to Engraft

    This phase II trial studies how well combination chemotherapy, total body irradiation, and donor stem cell transplant work in treating patients with hematopoietic malignancies including those that are challenging to engraft. Giving chemotherapy and 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. 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving high-dose cyclophosphamide after the transplant may stop this from happening.
    Location: Laura and Isaac Perlmutter Cancer Center at NYU Langone, New York, New York

  • Recombinant EphB4-HSA Fusion Protein and Azacitidine or Decitabine in Treating Patients with Relapsed or Refractory Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia Previously Treated with a Hypomethylating Agent

    This pilot phase II trial studies the side effects of recombinant EphB4-HSA fusion protein when given together with azacitidine or decitabine in treating patients with myelodysplastic syndrome, chronic myelomonocytic leukemia, or acute myeloid leukemia that has come back or has not responded to previous treatment with a hypomethylating agent. Recombinant EphB4-HSA fusion protein may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Hypomethylating agents, such as azacitidine and decitabine, slow down genes that promote cell growth and can kill cells that are dividing rapidly. Giving recombinant EphB4-HSA fusion protein together with azacitidine or decitabine may work better in treating patients with myelodysplastic syndrome, chronic myelomonocytic leukemia, or acute myeloid leukemia.
    Location: USC / Norris Comprehensive Cancer Center, Los Angeles, California


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