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 225
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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: 164 locations

  • Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation

    The purpose of this study is to determine if a search strategy of searching for an HLA-matched unrelated donor for allogeneic transplantation if possible then an alternative donor if an HLA-matched unrelated donor is not available versus proceeding directly to an alternative donor transplant will result in better survival for allogeneic transplant recipients within 2 years after study enrollment.
    Location: 24 locations

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

    This phase I trial studies the side effects and how well pevonedistat, azacitidine, fludarabine phosphate, and cytarabine work in treating patients with acute myeloid leukemia or myelodysplastic syndrome that has come back (relapsed) or has not responded to treatment (refractory). Pevonedistat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Chemotherapy drugs, such as azacitidine, 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 more than one drug (combination chemotherapy) and pevonedistat may work better in treating patients with acute myeloid leukemia or myelodysplastic syndrome.
    Location: 19 locations

  • Magrolimab Monotherapy or Magrolimab in Combination With Azacitidine in Patients With Hematological Malignancies

    This trial will evaluate magrolimab, a monoclonal antibody which is designed to block a protein called CD47, which is widely expressed on human cancer cells. Blocking CD47 with magrolimab may enable the body's immune system to find and destroy the cancer cells. In this study, magrolimab may be given alone or in combination with azacitidine to patients with acute myeloid leukemia (AML) or higher risk myelodysplastic syndrome (MDS). Azacitidine is a drug used for treatment of AML or MDS in patients who are not eligible for typical chemotherapy. The major aims of the study are: to confirm the safety and tolerability of magrolimab monotherapy in a relapsed / refractory AML and MDS population, and of magrolimab in combination with azacitidine in previously untreated AML and MDS; to evaluate the efficacy of magrolimab monotherapy in relapsed / refractory AML / MDS, and of magrolimab in combination with azacitidine in previously untreated AML / MDS, as measured by the objective response rate; and to evaluate the safety, tolerability, and efficacy of magrolimab monotherapy or combination with azacitidine in low-risk MDS patients as measured by RBC transfusion independence rate.
    Location: 18 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: 17 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. Giving azacitidine or decitabine before usual chemotherapy may change the genetics of the leukemia cell by priming it to be more sensitive to the chemotherapy that will follow in treating patients with acute myeloid leukemia.
    Location: 13 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: 12 locations

  • Testing the Safety and Efficacy of the Combination of the Antibody Pembrolizumab and Entinostat in Patients with Myelodysplastic Syndrome Who Are Not Responding to Hypomethylating Agents

    This phase Ib trial studies the side effects and best dose of entinostat when given together with pembrolizumab in treating patients with myelodysplastic syndrome after deoxyribonucleic acid (DNA) methyltransferase inhibitor (DNMTi) therapy failure. Entinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body’s immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving entinostat together with pembrolizumab may work better in treating patients with myelodysplastic syndrome after DNMTi therapy failure.
    Location: 18 locations

  • Safety Study of MGD006 in Relapsed / Refractory Acute Myeloid Leukemia (AML) or Intermediate-2 / High Risk MDS

    The primary goal of this Phase 1 / 2, dose-escalation study, is to determine the maximum tolerated dose level of flotetuzumab (MGD006) in patients with AML whose disease is not expected to benefit from cytotoxic chemotherapy. Studies will also be done to see how the drug acts in the body (pharmacokinetics [PK], pharmacodynamics) and to evaluate potential anti-tumor activity of flotetuzumab.
    Location: 14 locations

  • Study to Evaluate Imetelstat (GRN163L) 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: 10 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: 9 locations

  • Study of APVO436 in Patients With AML or MDS

    APVO436 is being studied in this Phase 1 / 1b, open-label, multi-center, dose-escalation study to evaluate the safety, pharmacokinetic / pharmacodynamic and clinical activity of APVO436 monotherapy in: 1) patients with AML that have relapsed on prior therapy or are refractory to therapy and are not candidates for intensive chemotherapy or transplant, and 2) patients with MDS that have > 5% blasts in the bone marrow or blasts in the peripheral blood who have also failed prior therapy with an hypomethylating agent (HMA). The primary objective of the Phase 1 part of the study is to determine the recommended dose of APVO436 administered intravenously to patients with AML or MDS. The primary objective of the Phase 1b part of the study is to evaluate the clinical activity of APVO436 in patients with AML or MDS.
    Location: 8 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 (relapsed) or does not respond to treatment (refractory). Immunotherapy with monoclonal antibodies, such as ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. 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: 10 locations

  • A Study Evaluating Venetoclax in Combination With Azacitidine in Participants 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 participants with treatment-naïve higher-risk MDS comprising a dose-escalation portion and a safety expansion portion.
    Location: 7 locations

  • Phase 1-2 Study of Low Dose ASTX727 (ASTX727 LD) in Lower Risk MDS

    Multicenter, open-label study of various ASTX727 LD doses and schedules to assess safety, pharmacodynamics, pharmacokinetics, and hematologic response in subjects with IPSS risk category of low-risk or Intermediate-1 MDS. This study will be conducted in two phases. In phase 1 subjects will be randomized into 3 cohorts in a 28-day cycles. Phase 2, 80 new subjects will be randomized in a 1:1 ratio into 2 doses / schedules.
    Location: 11 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

  • APR-246 in Combination With Azacitidine for TP53 Mutated AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes) Following Allogeneic Stem Cell Transplant

    A multi-center, open label, Phase II clinical trial to assess the safety and efficacy of APR-246 in combination with azacitidine as maintenance therapy after allogeneic HSCT (hematopoietic stem cell transplant) for patients with TP53 mutant AML or MDS.
    Location: 6 locations

  • Study to Investigate the Safety and Clinical Activity of GSK3326595 and Other Agents to Treat Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)

    GSK3326595 is a potent, selective, reversible inhibitor of the protein arginine methyltransferase 5 (PRMT5) / Methylosome protein 50 (MEP50) complex that is being tested as an oral treatment for human participants with cancer. Myelodysplastic syndrome and acute myeloid leukemia are bone marrow neoplasms for which novel, effective therapies are desperately needed. This is an open-label, multicenter, multi-part study to evaluate the safety, tolerability, and clinical activity of GSK3326595 in participants with relapsed and refractory MDS, chronic myelomonocytic leukemia (CMML), and hypoproliferative AML that has evolved from an antecedent MDS. The study will be conducted in two parts and at the end of Part 1, if pre-specified criteria are met, then the study will be expanded with three additional parts that will be opened in parallel (Part 2A, 2B and 2C). Part 1 is composed of a single-arm dose expansion cohort to determine the clinical benefit rate of GSK3326595. Part 2A is a randomized head-to-head Phase II evaluation of GSK3326595 compared to investigator's choice of best available care (BAC). Part 2B is composed of an abbreviated series of dose escalation cohorts followed by a single-arm dose expansion cohort to determine the overall response rate of the combination of GSK3326595 plus 5-azaciditine in newly-diagnosed MDS. Part 2C is a single-arm dose expansion study to evaluate the clinical activity of single-agent GSK3326595 in participants with AML whose disease contains mutations in spliceosome proteins.
    Location: 6 locations

  • Safety Study of Cord Blood Units for Stem Cell Transplants

    Background: - Cord blood is blood that is taken from the umbilical cord and placenta of healthy newborns after childbirth. The cord blood collected from a baby is called a cord blood unit. Cord blood units are stored frozen in public cord blood banks. About 10,000 cord blood transplants have been performed in children and adults for blood cancers and other diseases in the world. These transplants have helped save lives and improve treatments. However, not all available units of cord blood have been collected, stored, and licensed according to specific government requirements. These unlicensed units can still be used in transplant, but they can only be given as part of specific research studies. This study will evaluate the safety of giving these unlicensed units by recording any problems that may occur during and after giving the cord blood. Objectives: - To test the safety and effectiveness of unlicensed cord blood units in people who need stem cell transplants. Eligibility: - Individuals who are scheduled to have a stem cell transplant. Design: - Participants will be screened with a medical history and physical exam. - Participants will receive the cord blood unit as part of their stem cell transplant procedure. The transplant will be performed according to the current standard of care for the procedure. - After the transplant, participants will be monitored for up to 1 year. Any problems or side effects from the transplant will be treated as necessary. All outcomes will be reported to the National Cord Blood Program and to the Center for International Blood and Marrow Transplant.
    Location: 6 locations

  • AZD6738 for the Treatment of Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia Progressing on Standard Therapy

    This phase Ib trial studies the side effects, best dose, and response to AZD6738 in treating patients with myelodysplastic syndrome or chronic myelomonocytic leukemia progressing on standard therapy. DNA is the genetic material that serves as the body’s instruction book. Cancer is caused by changes (mutations) to genes (DNA) that control the way cells function. AZD6738 blocks a protein called ATR. ATR notices when there is injury to DNA and works to repair that damage. Studies done in a laboratory setting and cell lines suggest that myelodysplastic syndrome and chronic myelomonocytic leukemia cells rely specifically on the ATR pathway (a network of genes that interact with ATR) to fix DNA damage and survive; by inhibiting ATR with AZD6738, myelodysplastic syndrome or chronic myelomonocytic leukemia cells appear to selectively accumulate DNA damage and die, but healthy cells appear to be less sensitive to this drug. Inhibiting ATR may be a way to selectively target myelodysplastic syndrome or chronic myelomonocytic leukemia cells for treatment.
    Location: 5 locations

  • Durvalumab and Tremelimumab for Pediatric Malignancies

    The purpose of the study is to determine the recommended dose of durvalumab and tremelimumab (immunotherapy drugs) in pediatric patients with advanced solid and hematological cancers and expand in a second phase to test the efficacy of these drugs once this dose is determined.
    Location: 5 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 has failed primary therapy, that does not respond to treatment (refractory), or has come back (recurrent). Pevonedistat and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: 5 locations

  • A Dose-finding Study of CC-90009 in Subjects With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher-risk Myelodysplastic Syndromes

    CC-90009-AML-001 is a phase 1, open-label, dose escalation and expansion, study in subjects with relapsed or refractory acute myeloid leukemia and relapsed or refractory high-risk myelodysplastic syndrome.
    Location: 5 locations

  • A Study of ASTX030 (Cedazuridine in Combination With Azacitidine) in MDS, CMML, or AML

    Study ASTX030-01 is designed to move efficiently from Phase 1 to Phase 3. Phase 1 consists of an open-label Dose Escalation Stage (Stage A) using multiple cohorts at escalating dose levels of oral cedazuridine and azacitidine (only one study drug will be escalated at a time) followed by a Dose Expansion Stage (Stage B) of ASTX030. Phase 2 is a randomized open-label crossover study to compare oral ASTX030 to subcutaneous (SC) azacitidine. Phase 3 is a randomized open-label crossover study comparing the final oral ASTX030 tablet to SC azacitidine. The duration of the study is expected to be approximately 36 months.
    Location: 4 locations

  • A Study of JNJ-67571244 in Participants With Relapsed or Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

    The main purpose of this study are to determine the recommended Phase 2 dose(s) (RP2D) route of administration, schedule and the maximum tolerated dose (MTD) in Part 1 and to determine the safety and tolerability of JNJ-67571244 at the RP2D regimen(s) and to evaluate the preliminary clinical activity of JNJ-67571244 in Part 2.
    Location: 6 locations


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