Treatment Clinical Trials for Myelodysplastic Syndrome

Clinical trials are research studies that involve people. The clinical trials on this list are for myelodysplastic syndrome 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 214
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  • Azacitidine with or without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients with Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

    This randomized phase II / III trial studies how well azacitidine with or without nivolumab or midostaurin, or decitabine and cytarabine alone work in treating older patients with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome. Drugs used in chemotherapy, such as azacitidine, decitabine, 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. Monoclonal antibodies, such as nivolumab, may interfere with the ability of cancer cells to grow and spread. Midostaurin may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving azacitidine with or without nivolumab or midostaurin, or decitabine and cytarabine alone may kill more cancer cells.
    Location: 419 locations

  • 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: 149 locations

  • Allo vs Hypomethylating / Best Supportive Care in MDS (BMT CTN 1102)

    This study is designed as a multicenter trial, with biological assignment to one of two study arms; Arm 1: Reduced intensity conditioning allogeneic hematopoietic cell transplantation (RIC-alloHCT), Arm 2: Non-Transplant Therapy / Best Supportive Care.
    Location: 23 locations

  • Controlled Study of Rigosertib Versus Physician's Choice of Treatment in MDS Patients After Failure of an HMA

    The study's primary objective [in a population of patients with MDS after failure of treatment with azacitidine (AZA) or decitabine (DAC)], is to compare the overall survival (OS) of patients in the rigosertib group vs the Physician's Choice group, in all patients and in a subgroup of patients with IPSS-R very high risk.
    Location: 18 locations

  • Open-label Study of FT-2102 With or Without Azacitidine or Cytarabine in Patients With AML or MDS With an IDH1 Mutation

    This Phase 1 / 2 study will evaluate the safety, efficacy, PK, and PD of FT-2102 as a single agent or in combination with azacitidine or cytarabine. The Phase 1 stage of the study is split into 2 distinct parts: a dose escalation part, which will utilize an open-label design of FT-2102 (single agent) and FT-2102 + azacitidine (combination agent) administered via one or more intermittent dosing schedules followed by a dose expansion part. The dose expansion part will enroll patients in up to 5 expansion cohorts, exploring single-agent FT-2102 activity as well as combination activity with azacitidine or cytarabine. Following the completion of Phase 1, Phase 2 will begin enrollment. Patients will be enrolled across 6 different cohorts, examining the effect of FT-2102 (as a single agent) and FT-2102 + azacitidine (combination) on various AML / MDS disease states.
    Location: 13 locations

  • A Biomarker-Directed Phase 2 Trial of SY-1425 in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

    The purpose of this study is to determine the activity of SY-1425 in relapsed / refractory acute myeloid leukemia (AML) patients, relapsed / refractory higher-risk myelodysplastic syndrome (MDS) patients (SY-1425 administered as a monotherapy or in combination with daratumumab), newly diagnosed treatment naïve AML patients who are unlikely to tolerate standard intensive chemotherapy (SY-1425 administered as a monotherapy or in combination with azacitidine), or lower-risk myelodysplastic syndrome (MDS) patients (SY-1425 administered as a monotherapy) who are positive for a RARA biomarker.
    Location: 12 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

  • 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

  • Phase 1 Study to Evaluate MEDI4736 in Subjects With Myelodysplastic Syndrome

    This is a multicenter, open-label, Phase 1 study to assess the safety and antitumor activity of MEDI4736 as Monotherapy or in Combination with Tremelimumab with or without Azacitidine in Subjects with myelodysplastic syndrome after treatment with hypomethylating agents
    Location: 10 locations

  • A Study Evaluating Venetoclax in Combination With Azacitidine in Subjects With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

    This is a Phase 1b, open-label, non-randomized, multicenter, dose-finding study evaluating venetoclax in combination with azacitidine in subjects with treatment-naïve higher-risk MDS comprising a dose-escalation portion and a safety expansion portion.
    Location: 10 locations

  • Ipilimumab and Decitabine in Treating Patients with Relapsed or Refractory Myelodysplastic Syndrome or Acute Myeloid Leukemia

    This phase I trial studies the side effects and best dose of ipilimumab when given together with decitabine in treating patients with myelodysplastic syndrome or acute myeloid leukemia that has returned after a period of improvement or does not respond to treatment. Monoclonal antibodies, such as ipilimumab, may block cancer growth in different ways by targeting certain cells. 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 ipilimumab and decitabine may work better in treating patients with relapsed or refractory myelodysplastic syndrome or acute myeloid leukemia.
    Location: 9 locations

  • Outpatient Induction Chemotherapy in Treating Patients with Acute Myeloid Leukemia or Advanced Myelodysplastic Syndrome

    This pilot clinical trial studies the feasibility of having induction chemotherapy in an outpatient setting. Patients with acute leukemia (AML) or advanced myelodysplastic syndrome (MDS), at least 18 years of age will be examined. Treating eligible patients with induction chemotherapy in an outpatient setting may save in healthcare cost and improve a patients’ quality of life.
    Location: 9 locations

  • A Safety and Efficacy Study of Pracinostat and Azacitidine in Patients With High Risk Myelodysplastic Syndromes

    This is a Phase 2, two-stage study of the safety and efficacy of pracinostat in combination with azacitidine in patients with IPSS-R high and very high risk MDS who are previously untreated with HMAs. Enrollment in this study will be limited to high / very high risk MDS because this group represents the highest unmet need, with median survival of less than 18 months. Stage 1 will be conducted as an open-label single arm in up to 40 subjects to assess if this regimen with a lower pracinostat dose results in a discontinuation rate that meets a predefined threshold and in efficacy that justifies expansion of enrollment into Stage 2. A discontinuation rate of approximately ≤10% in Stage 1, a rate comparable to that observed with azacitidine alone in study MEI-003, is considered desirable and supports expansion into Stage 2. Stage 2 will be conducted as a randomized, double-blind, placebo-controlled, two-arm design to confirm that the discontinuation rate observed in an open-label design can be reproduced in a placebo controlled design, and to provide unbiased information on efficacy and safety. Subjects will be randomized in a 1:1 ratio to one of the 2 treatment arms (Arm A, pracinostat + azacitidine and Arm B, placebo + azacitidine). Study drugs should be continued until disease progression or intolerable toxicity. It is important to note that the median time to achieving a response with azacitidine alone is 4 to 5 months. Furthermore, the median time to achieving a CR in study MEI-003 was 4 cycles. Therefore, early (<6 months) discontinuation of trial therapy for 'no response' should be avoided. The Medical Monitor should be contacted prior to discontinuing a subject from the study to discuss the rationale for discontinuation.
    Location: 8 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 (BAML-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: 9 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: 10 locations

  • Azacitidine or Decitabine in Epigenetic Priming in Patients with Newly Diagnosed Acute Myeloid Leukemia

    This randomized phase II trial studies how well azacitidine or decitabine work in epigenetic priming in patients with newly diagnosed acute myeloid leukemia. Azacitidine and decitabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: 7 locations

  • HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide

    This is a multi-center, single arm Phase II study of hematopoietic cell transplantation (HCT) using human leukocyte antigen (HLA)-mismatched unrelated bone marrow transplantation donors and post-transplantation cyclophosphamide (PTCy), sirolimus and mycophenolate mofetil (MMF) for graft versus host disease (GVHD) prophylaxis in patients with hematologic malignancies.
    Location: 7 locations

  • Panobinostat, Fludarabine Phosphate, and Cytarabine in Treating Younger Patients with Refractory or Relapsed Acute Myeloid Leukemia or Myelodysplastic Syndrome

    This phase I trial studies the side effects and best dose of panobinostat when given together with fludarabine phosphate and cytarabine in treating younger patients with acute myeloid leukemia or myelodysplastic syndrome that has not responded to previous treatment (refractory) or has returned after a period of improvement (relapsed). Panobinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as fludarabine phosphate 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. Giving panobinostat together with fludarabine phosphate and cytarabine may kill more cancer cells.
    Location: 7 locations

  • A Study of CC-90002 in Subjects With Acute Myeloid Leukemia (AML) and High-risk Myelodysplastic Syndrome (MDS)

    Study CC-90002-AML-001 is an open-label, Phase 1 dose escalation (Part A) and expansion (Part B), clinical study of CC-90002, administered by intravenous (IV) infusion, in subjects with relapsed and / or primary refractory AML and high-risk MDS. The study will explore escalating doses of CC-90002 using a 3 + 3 dose escalation design in Part A, followed by dose expansion in Part B. The primary objective is to determine the safety and tolerability of CC-90002 and also to define the non-tolerated dose (NTD), the maximum tolerated dose (MTD) and / or the recommended Phase 2 dose (RP2D) of CC-90002.
    Location: 7 locations

  • Study to Evaluate Imetelstat (JNJ-63935937) in Subjects With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

    The purpose of this study is to evaluate the efficacy and safety of imetelstat in transfusion dependent participants with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed / refractory to erythropoiesis-stimulating agent (ESA) treatment.
    Location: 9 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

  • Low Dose Azacitidine after Transplant in Preventing Recurrence in Patients with Myelodysplastic Syndromes or Acute Myeloid Leukemia in Remission

    This phase II trial studies the side effects and how well low dose azacitidine after transplant works in preventing cancer from coming back in patients with myelodysplastic syndromes or acute myeloid leukemia in remission. Drugs used in chemotherapy, such as azacitidine, work to stop the growth of cancer cells either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
    Location: 6 locations

  • Selinexor, Fludarabine Phosphate, and Cytarabine in Treating Younger Patients with Refractory or Relapsed Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, or Myelodysplastic Syndromes

    This pilot phase I / II trial studies the side effects and best dose of selinexor when given together with fludarabine phosphate and cytarabine in treating younger patients with acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndromes that did not go into remission after treatment (refractory) or has come back after treatment (relapsed). One way cancer cells continue to grow by escaping from mechanisms that normally control human cell growth, such as a type of protein called a tumor suppressor protein. Tumor suppressor proteins normally cause cancer cells to die. Selinexor works by trapping tumor suppressor proteins within the cancer cells, causing them to stop growing or die. Fludarabine phosphate and cytarabine are drugs used in chemotherapy that stop cancer cells from dividing. Giving selinexor with fludarabine phosphate and cytarabine may work better in treating acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndromes in younger patients.
    Location: 5 locations

  • A Study Evaluating Venetoclax Alone and in Combination With Azacitidine in Subjects With Relapsed / Refractory Myelodysplastic Syndromes (MDS)

    This is a Phase 1b, dose-ranging, open-label, multicenter study designed to evaluate the safety and pharmacokinetics of venetoclax as a single-agent and in combination with azacitidine in participants with relapsed / refractory Myelodysplastic Syndromes (MDS).
    Location: 8 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, chronic myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.
    Location: 5 locations


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