Treatment Clinical Trials for Myeloproliferative Neoplasm

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Clinical trials are research studies that involve people. The clinical trials on this list are for myeloproliferative neoplasm 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 118
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  • Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients with Down Syndrome

    This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome. Drugs used in chemotherapy 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. Response-based chemotherapy separates patients into different risk groups and treats them according to how they respond to the first course of treatment (Induction I). Response-based treatment may be effective in treating acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome while reducing the side effects.
    Location: 142 locations

  • PEG-interferon Alfa-2a in Treating Patients With High Risk Polycythemia Vera or High Risk Essential Thrombocythemia

    The purpose of this study is to look at the effectiveness of giving patients who have been diagnosed with essential thrombocythemia (ET) and polycythemia vera (PV) a study drug regimen using aspirin (acetylsalicylic acid) and pegylated interferon alfa-2a (PEG-interferon alfa-2a) (instead of the standard treatment drug called hydroxyurea), for whom this drug may not be suitable. The drug may not be suitable either because it is not adequately controlling the number of blood cells or some specific side effects occur.
    Location: 20 locations

  • Ruxolitinib Phosphate Before Reduced Intensity Donor Stem Cell Transplant in Treating Patients with Myelofibrosis

    This phase II trial studies how well adding ruxolitinib phosphate before a reduced intensity donor stem cell transplant works in treating patients with myelofibrosis. Ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving ruxolitinib phosphate and chemotherapy before a donor stem cell may help 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.
    Location: 11 locations

  • PEG-Interferon Alfa-2a or Hydroxyurea in Treating Patients With High Risk Polycythemia Vera or High Risk Essential Thrombocythemia

    This research looks at two conditions, essential thrombocythemia (ET) and polycythemia vera (PV). ET causes people to produce too many blood cells called platelets and PV causes too many platelets and red blood cells to be made. The purpose of this study is to look at the effectiveness of giving participants who have been diagnosed with ET or PV one of two different study regimens over time. The study regimens are either “PEGASYS” (or pegylated interferon alfa-2a [PEG interferon alfa-2a]) or “aspirin and hydroxyurea” (also called hydroxycarbamide). Patients may be a newly diagnosed subject or a subject already receiving treatment for either PV or ET. Each of the study drugs used in this study is already being used to treat subjects with ET or PV currently but we are unsure which study drug is better.
    Location: 15 locations

  • Ponatinib in Patients With Resistant Chronic Phase Chronic Myeloid Leukemia (CML) to Characterize the Efficacy and Safety of a Range of Doses

    The purpose of this study is to compare and characterize the efficacy and safety of ponatinib in patients with resistant chronic myeloid leukemia (CML) in chronic phase (CP) in a range of doses.
    Location: 14 locations

  • Ipilimumab or Nivolumab in Treating Patients with Relapsed Hematologic Malignancies after Donor Stem Cell Transplant

    This phase I / Ib trial studies the side effects and best dose of ipilimumab or nivolumab in treating patients with cancers of the blood and blood-forming tissues (hematologic cancers) that have returned after a period of improvement (relapsed) after donor stem cell transplant. Monoclonal antibodies, such as ipilimumab and nivolumab, may interfere with the ability of cancer cells to grow and spread.
    Location: 12 locations

  • Lestaurtinib in Treating Patients With Myelofibrosis

    The purpose of this study is to find out how effective CEP-701 is at controlling the protein JAK2. There is a protein that is found to be present in the majority of myelofibrosis patients (JAK2) and the investigational drug CEP-701 is being studied to see if it will stop this protein from functioning and thereby help control the disease.
    Location: 11 locations

  • A Study of INCB050465 in Combination With Ruxolitinib in Subjects With Myelofibrosis

    The purpose of this study is to evaluate the safety, tolerability, and efficacy of the combination of INCB050465 and ruxolitinib in subjects with myelofibrosis.
    Location: 12 locations

  • Selinexor in Treating Younger Patients with Relapsed or Refractory Acute Lymphoblastic Leukemia or Acute Myeloid Leukemia

    This phase I trial studies the side effects and the best dose of selinexor in treating younger patients with acute lymphoblastic leukemia or acute myeloid leukemia that has returned or has become resistant to standard therapies. Selinexor may prevent leukemia cells from growing and may lead to the destruction of leukemia cells.
    Location: 10 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 and Decitabine in Treating Patients with Myeloproliferative Neoplasms

    This phase I / II trial studies the side effects and best dose of ruxolitinib phosphate when given together with decitabine and to see how well they work in treating patients with myeloproliferative neoplasms, a group of diseases of the bone marrow in which excess cells are produced. Ruxolitinib phosphate may stop the growth of cancer cells by blocking one of the proteins needed for cell growth. 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. Giving ruxolitinib phosphate with decitabine may be an effective treatment for myeloproliferative neoplasms.
    Location: 10 locations

  • Donor Umbilical Cord Blood Transplant with or without Ex-vivo Expanded Cord Blood Progenitor Cells in Treating Patients with Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, or Myelodysplastic Syndromes

    This randomized phase II trial studies how well donor umbilical cord blood transplant with or without ex-vivo expanded cord blood progenitor cells works in treating patients with acute myeloid leukemia, acute lymphoblastic leukemia, chronic myelogenous leukemia, or myelodysplastic syndromes. Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's cells. When the healthy stem cells and ex-vivo expanded cord blood progenitor cells 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 donor umbilical cord blood transplant plus ex-vivo expanded cord blood progenitor cells is more effective than giving a donor umbilical cord blood transplant alone.
    Location: 9 locations

  • Ruxolitinib (INCB018424) in Subjects With Primary Myelofibrosis, Post Essential Thrombocythemia-myelofibrosis and Post Polycythemia Vera-myelofibrosis

    To evaluate the effects of treatment with ruxolitinib (INCB018424) on spleen volume, symptoms and potential side effects in patients with PMF, PPV-MF and PET-MF who have platelet counts of 50 x 10^9 / L to 100 x 10^9 / L. It is anticipated that individualized dose optimization from the starting ruxolitinib level of 5 mg bid will be associated with reductions in splenomegaly, MF-associated symptoms and inflammatory cytokine levels.
    Location: 8 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

  • Ruxolitinib and Thalidomide in Treating Patients with Newly Diagnosed, Relapsed, or Refractory Myelofibrosis

    This phase II trial studies how well ruxolitinib and thalidomide work in treating patients with myelofibrosis that is newly diagnosed, has come back, or does not respond to treatment. Ruxolitinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Thalidomide may shrink cancer and reduce the number of blood transfusion needed. Giving ruxolitinib and thalidomide may work better in treating patients with myelofibrosis.
    Location: 7 locations

  • Open-Label, Dose-Escalation Study of INCB054828 in Subjects With Advanced Malignancies

    The purpose of this study will be to evaluate the safety, tolerability, and pharmacological activity of INCB054828 in subjects with advanced malignancies. This study will have three parts, dose escalation (Part 1), dose expansion (Part 2) and combination therapy (Part 3).
    Location: 6 locations

  • Dose-Finding Study of Pacritinib in Patients With Thrombocytopenia and Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis Previously Treated With Ruxolitinib

    This is a Bayesian adaptive dose-finding study in patients with primary or secondary myelofibrosis: 1. who have failed therapy with ruxolitinib on the basis of intolerance or loss of efficacy, 2. are thrombocytopenic (platelet count of ≤100,000 / μL), 3. highly symptomatic (DIPSS risk score of Intermediate-1 to High Risk), 4. and have splenomegaly (assessed by physical examination). The study is designed to support a pacritinib dosage selection decision. Three dosages will be evaluated, with patients randomized 1:1:1 to pacritinib 100 mg quaque die (QD), pacritinib 100 mg bis in die (BID), or pacritinib 200 mg bis in die (BID). Assigned treatment will continue for 24 weeks unless the patient experiences progressive disease, intolerable AEs, withdraws consent, or until the assigned treatment arm is closed. All patients should complete all visit procedures through Week 24, including patients who stop pacritinib treatment or have protocol-defined progressive disease prior to Week 24, unless patient withdraws consent, dies, undergoes splenic irradiation or splenectomy, or initiates any nonprotocol-directed anti-myelofibrosis treatment. The dosage selection process will be based on pre-specified efficacy and safety parameters, including model-based dose-response. The maximum duration of trial participation for an individual patient will be approximately 7 months. The estimated duration of the entire study is approximately 2 years if the maximum number of patients are enrolled.
    Location: 11 locations

  • Phase 2 Study of Ruxolitinib Versus Anagrelide in Subjects With Essential Thrombocythemia Who Are Resistant to or Intolerant of Hydroxyurea (RESET-272)

    The purpose of this study is to evaluate the efficacy and safety of ruxolitinib versus anagrelide in subjects with essential thrombocythemia who are resistant to or intolerant of hydroxyurea.
    Location: 6 locations

  • Study of Biomarker-Based Treatment of Acute Myeloid Leukemia

    This screening and multi-sub-study Phase 1b / 2 trial will establish a method for genomic screening followed by assigning and accruing simultaneously to a multi-study "Master Protocol (BMAL-16-001-M1)." The specific subtype of acute myeloid leukemia will determine which sub-study, within this protocol, a participant will be assigned to evaluate investigational therapies or combinations with the ultimate goal of advancing new targeted therapies for approval. The study also includes a marker negative sub-study which will include all screened patients not eligible for any of the biomarker-driven sub-studies.
    Location: 5 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

  • Vosaroxin and Cytarabine in Treating Patients with Untreated Acute Myeloid Leukemia

    This phase II trial studies how well vosaroxin and cytarabine work in treating patients with untreated acute myeloid leukemia. Drugs used in chemotherapy, such as vosaroxin and 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: 5 locations

  • Alisertib in Combination with Cytarabine and Idarubicin in Treating Patients with High-Risk Acute Myeloid Leukemia

    This phase II trial studies how well alisertib, in combination with cytarabine and idarubicin, works in treating patients with high-risk acute myeloid leukemia. Drugs used in chemotherapy, such as cytarabine and idarubicin, 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. Alisertib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving alisertib together with cytarabine and idarubicin may kill more cancer cells.
    Location: 4 locations

  • Sirolimus, Cyclosporine, and Mycophenolate Mofetil in Preventing Graft-versus-Host Disease in Treating Patients with Blood Cancer Undergoing Donor Peripheral Blood Stem Cell Transplant

    This phase II trial studies how well sirolimus, cyclosporine and mycophenolate mofetil works in preventing graft-vs-host disease (GVHD) in patients with blood cancer undergoing donor peripheral blood stem cell (PBSC) transplant. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving total-body irradiation together with sirolimus, cyclosporine, and mycophenolate mofetil before and after transplant may stop this from happening.
    Location: 3 locations

  • PH 1 Study to Evaluate Safety and Tolerability of XmAb14045 in Patients With CD123-expressing Hematologic Malignancies

    The purpose of this study is to determine the safety and tolerability of weekly intravenous (IV) administration of XmAb14045 and to determine the maximally tolerated dose (MTD) after the first dose, and then to determine the MTD after second and subsequent infusions.
    Location: 3 locations

  • Transplantation of Ex Vivo Expanded, UCB-derived, Stem & Progenitor Cells vs. Unmanipulated UCB for HM Patients

    This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome or chronic myeloid leukemia, all with required disease features rendering them eligible for allogeneic transplantation.
    Location: 3 locations


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