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 71
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  • Chemotherapy before and after Donor Bone Marrow Transplant in Treating Younger Patients with Hematologic Cancer

    This pilot phase II trial studies how well chemotherapy before and after a donor bone marrow transplant works in treating younger patients with hematologic cancer. Giving chemotherapy and total-body irradiation before a donor bone marrow 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 related donor, that do not exactly match the patient’s blood, are infused into the patient they may replace the patient’s immune cells and help destroy any remaining cancer cells. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells called graft-versus-host disease. Giving cyclophosphamide after the transplant may stop this from happening.
    Location: 14 locations

  • 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: 14 locations

  • Guadecitabine (SGI-110) vs Treatment Choice in Adults With MDS or CMML Previously Treated With HMAs

    A Phase 3, randomized, open-label, parallel-group, multicenter study designed to evaluate the efficacy and safety of guadecitabine in subjects with MDS or CMML who failed or relapsed after adequate prior treatment with azacitidine, decitabine, or both. This global study will be conducted in approximately 15 countries. Approximately 408 subjects from approximately 100 study centers will be randomly assigned in a 2:1 ratio to either guadecitabine (approximately 272 subjects) or Treatment Choice (approximately 136 subjects). The study consists of a 14-day screening period, a treatment period, a safety follow-up visit, and a long-term follow-up period. The study is expected to last more than 2 years, and the duration of individual subject participation will vary. Subjects may continue to receive treatment for as long as they continue to benefit.
    Location: 11 locations

  • SL-401 in Advanced, High Risk Myeloproliferative Neoplasms (Systemic Mastocytosis, Advanced Symptomatic Hypereosinoophic 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 hypereosinoophic 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 18 patients with each of the 2 myeloproliferative malignancies: MF and CMML. In entirety, the Stage 2 portion will consist of up to 36 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: 11 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 1 Study Evaluating CPI-0610 in Patients With Acute Leukemia, Myelodysplastic Syndrome, Myelodysplastic / Myeloproliferative Neoplasms, and Myelofibrosis

    Open-label, sequential dose escalation and expansion study of CPI-0610 in patients with previously treated Acute Leukemia, Myelodysplastic Syndrome, Myelodysplastic / Myeloproliferative Neoplasms, and Myelofribrosis. CPI-0610 is a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins.
    Location: 5 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: 6 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

  • 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: 4 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

  • Targeted Therapy in Treating Patients with Relapsed or Refractory Acute Lymphoblastic Leukemia or Acute Myelogenous Leukemia

    This phase II trial studies how well targeted therapy works in treating patients with acute lymphoblastic leukemia or acute myelogenous leukemia that has come back after a period of improvement or does not respond to treatment. Testing patients' blood or bone marrow to find out if their type of cancer may be sensitive to a specific drug may help doctors choose more effective treatments. Dasatinib, nilotinib, sunitinib malate, sorafenib tosylate, and ponatinib hydrochloride may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving targeted therapy based on cancer type may be an effective treatment for acute lymphoblastic leukemia or acute myelogenous 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

  • TGR-1202 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 phosphoinositide-3-kinase (PI3K)-delta inhibitor TGR-1202 (TGR-1202) 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. PI3K-delta inhibitor TGR-1202 and ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: 4 locations

  • A Combination Study of PF-04449913 (Glasdegib) and Azacitidine In 1st Line MDS, AML and CMML Patients

    This multi center randomized (1:1), double blind, placebo controlled Phase 1b / 2 study is designed to compare the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of PF 04449913 or placebo when combined with azacitidine in patients with previously untreated Intermediate 2 or High Risk Myelodysplastic Syndrome (MDS), Acute Myeloid Leukemia (AML) with 20-30% blasts and multi lineage dysplasia, and Chronic Myelomonocytic Leukemia (CMML). This clinical study includes two components: (a) a Phase 1b safety lead in and (b) a randomized Phase 2.
    Location: 5 locations

  • Pomalidomide after Combination Chemotherapy in Treating Patients with Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

    This phase I trial studies the side effects and best dose of pomalidomide after combination chemotherapy in treating patients with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome. Drugs used in chemotherapy, such as cytarabine, daunorubicin hydrochloride, and etoposide, 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. Pomalidomide may kill cancer cells by stopping blood flow to the cancer and by stimulating white blood cells to kill cancer cells. Giving more than one drug (combination chemotherapy) and pomalidomide may kill more cancer cells.
    Location: 3 locations

  • Phase II Part 2 Expansion of Oral Rigosertib in Combination With Azacitidine

    This study, is a Phase I / II clinical trial in three parts: Phase I Dose Escalation, Phase II, Part 1 RPTD Cohort, and Phase II, Part 2 Expansion. The first two parts have been completed. The Phase II, Part 2 Expansion will assess if treatment with rigosertib in combination with azacitidine, has measurable effects in patients with myelodysplastic syndrome (MDS). Safety of patients is an objective throughout all parts of the study.
    Location: 3 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

  • 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: Vanderbilt University / Ingram Cancer Center, Nashville, Tennessee

  • 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

  • 211^At-BC8-B10 before Donor Stem Cell Transplant in Treating Patients with High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, or Myelodysplastic Syndrome

    This phase I / II clinical trial studies the side effects and best dose of 211^astatine(At)-BC8-B10 before donor stem cell transplant in treating patients with high-risk acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndrome. Radioactive substances, such as astatine-211, linked to monoclonal antibodies, such as BC8, can bind to cancer cells and give off radiation which may help kill cancer cells and have less of an effect on healthy cells before donor stem cell transplant.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • 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: Moffitt Cancer Center, Tampa, Florida


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