Treatment Clinical Trials for Myelodysplastic Syndrome

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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 208
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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: 143 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: 19 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

  • 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

  • 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, 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 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

  • 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

  • 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: 11 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

  • 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

  • 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: 8 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

  • Dose-Escalation Study of FT-2102 as a Single Agent and in Combination With Azacitidine in Patients With AML or MDS With an IDH1 Mutation

    This Phase 1 / 1b study will utilize a multicenter, open-label dose-escalation design to evaluate the safety, PK, and PD of FT-2102 (single agent) and FT-2102 + azacitidine (combination agent) administered via one or more intermittent dosing schedules. Approximately 48 patients will be enrolled in the dose-escalation portion of this study in one or more schedules followed by approximately 14 patients in expansion cohorts
    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

  • 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: 6 locations

  • Low Dose Cytarabine and Lintuzumab-Ac225 in Older AML Patients

    The study is a multicenter, open label Phase I / II trial. The goal of the Phase I part of this study is to find the highest tolerable dose of Lintuzumab-Ac225 that can be given with cytarabine to patients with AML. The goal of the Phase II part of this study is to learn if Lintuzumab-Ac225 and cytarabine can control AML. The safety of this drug combination will also be studied. Lintuzumab-Ac225 is designed to deliver radiation therapy directly inside leukemia cells without giving any radiation to the surrounding normal cells Cytarabine is designed to insert itself into DNA (genetic material) of cancer cells and stop the DNA from repairing itself.
    Location: 7 locations

  • A Study of Atezolizumab Administered Alone or in Combination With Azacitidine in Participants With Myelodysplastic Syndromes

    This is a multicenter, open-label, Phase 1b study of atezolizumab (anti-programmed death-ligand 1 [anti-PD-L1] monoclonal antibody) in participants who have hypomethylating agent (HMA)-naïve myelodysplastic syndromes (MDS) and are International Prognostic Scoring System-Revised (IPSS-R) intermediate / high / very high-risk, or have MDS relapsed or are refractory (R / R) to prior HMA therapy. The primary objectives of this study are to determine the safety and tolerability of atezolizumab therapy in these participant populations, including treatment in combination with azacitidine.
    Location: 7 locations

  • Safety and Efficacy Study of CC-486 and Durvalumab in Subjects With Myelodysplastic Syndromes

    Evaluate the safety and efficacy of CC-486 and Durvalumab in Subjects with Myelodysplastic Syndromes who failed to achieve an objective response post iHMA treatment
    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

  • 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

  • The Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo and Best Supportive Care in Subjects With Red Blood Cell (RBC) Transfusion-Dependent Anemia and Thrombocytopenia Due to International Prognostic Scoring System (IPSS) Low Risk Myelodysplastic Syndrome (MDS)

    Evaluation of the Efficacy and Safety of Oral Azacitidine plus Best Supportive care versus Placebo and Best Supportive care in subjects with red blood cell (RBC) transfusion-dependent anemia and thrombocytopenia due to International Prognostic Scoring System (IPSS) lower risk myelodysplastic syndromes (MDS).
    Location: 7 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

  • Bioequivalence & Food Effect Study in Patients With Solid Tumor or Hematologic Malignancies

    This is a Phase 1, open-label, multicenter, randomized, 2-stage crossover study consisting of 2 phases: Stage I - Pharmacokinetics (Bioequivalence), with an Extension Stage II - Pharmacokinetics (Food Effect) with an Extension This study will enroll approximately 60 subjects in stage I and 60 subjects in stage II with hematologic or solid tumor malignancies, excluding gastrointestinal tumors and tumors that have originated or metastasized to the liver for which no standard treatment exists or have progressed or recurred following prior therapy. Subjects must not be eligible for therapy of higher curative potential where an alternative treatment has been shown to prolong survival in an analogous population. Approximately 23 sites in the US and 2 in Canada will participate in this study.
    Location: 5 locations


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