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 51-75 of 195
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  • Dose Escalation and Proof-of-Concept Studies of Vactosertib (TEW-7197) Monotherapy in Patients With MDS

    This is a prospective, open-label, multicenter, phase 1 / 2 study of TEW-7197 in patients with low and intermediate risk of myelodysplastic syndrome (MDS).
    Location: 2 locations

  • Sertraline in Treating Participants with Low-Risk Myelodysplastic Syndrome

    This early phase I trial studies how well sertraline works in treating participants with low-risk myelodysplastic syndrome. Myelodysplastic syndrome is associated with bone marrow failure, which is caused by severe bone marrow inflammation. Sertraline may improve myelodysplastic syndrome by reducing inflammation in the bone marrow.
    Location: 2 locations

  • Donor Partial Immune Cell Depletion for the Treatment of Hematologic Malignancies

    This trial studies how well donor stem cell transplantation with alpha / beta T cell and B cell depletion (partial Immune Cell Depletion) works in treating patients with hematologic malignancies. Alpha / beta T cell and B cell depletion is a new method of cell processing for stem cell transplants with an unrelated donor or partially matched related donor using the CliniMACS device. There is a higher rate of complications using cells from an unrelated or partially matched related donor. T cells within the donor cells may cause a complication called graft versus host disease, where the transplanted cells from a donor can attack the body's normal cells. Donated B cells can sometimes be infected with a virus (Epstein Bar Virus or EBV) which may result in the development of enlarged lymph nodes (lymphoproliferative disorder). Alpha / beta T cell and B cell depletion may reduce some of the complications of the transplant and decrease the time it takes for the new stem cells to grow in the body.
    Location: 2 locations

  • Selective Depletion of CD45RA+ T Cells from Allogeneic Peripheral Blood Stem Cell Grafts from HLA-Matched Related and Unrelated Donors in Preventing GVHD

    This phase II trial is for patients with acute lymphocytic leukemia, acute myeloid leukemia, myelodysplastic syndrome or chronic myeloid leukemia who have been referred for a peripheral blood stem cell transplantation to treat their cancer. In these transplants, chemotherapy and total-body radiotherapy ('conditioning') are used to kill residual leukemia cells and the patient's normal blood cells, especially immune cells that could reject the donor cells. Following the chemo / radiotherapy, blood stem cells from the donor are infused. These stem cells will grow and eventually replace the patient's original blood system, including red cells that carry oxygen to our tissues, platelets that stop bleeding from damaged vessels, and multiple types of immune-system white blood cells that fight infections. Mature donor immune cells, especially a type of immune cell called T lymphocytes (or T cells) are transferred along with these blood-forming stem cells. T cells are a major part of the curative power of transplantation because they can attack leukemia cells that have survived the chemo / radiation therapy and also help to fight infections after transplantation. However, donor T cells can also attack a patient's healthy tissues in an often-dangerous condition known as Graft-Versus-Host-Disease (GVHD). Drugs that suppress immune cells are used to decrease the severity of GVHD; however, they are incompletely effective and prolonged immunosuppression used to prevent and treat GVHD significantly increases the risk of serious infections. Removing all donor T cells from the transplant graft can prevent GVHD, but doing so also profoundly delays infection-fighting immune reconstitution and eliminates the possibility that donor immune cells will kill residual leukemia cells. Work in animal models found that depleting a type of T cell, called naive T cells or T cells that have never responded to an infection, can diminish GVHD while at least in part preserving some of the benefits of donor T cells including resistance to infection and the ability to kill leukemia cells. This clinical trial studies how well the selective removal of naive T cells works in preventing GVHD after peripheral blood stem cell transplants. This study will include patients conditioned with high or medium intensity chemo / radiotherapy who can receive donor grafts from related or unrelated donors.
    Location: 2 locations

  • Multi-institutional Prospective Research of Expanded Multi-antigen Specifically Oriented Lymphocytes for the Treatment of VEry High Risk Hematopoietic Malignancies

    This Phase I dose-escalation trial is designed to evaluate the safety of administering rapidly-generated multi-antigen-specific T lymphocytes, to HSCT recipients (Arm A) or future HSCT recipients (Arm B) for the treatment or relapsed or refractory hematopoietic malignancies, to determine if event-free survival (EFS) at six months after HSCT is improved with TAA-L administration at six months after HSCT for acute myeloid leukemia and MDS (Arm C).
    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

  • A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

    This is an open-label, dose escalation study to evaluate the safety, toxicity, and pharmacokinetics (PK) as well as preliminary efficacy of BTX-A51 capsules in participants with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The study will be done in two phases. Phase 1a of this study is designed to determine the dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD) of orally administered BTX-A51 in up to 35 participants who are evaluable for toxicity. Once the MTD is determined, it is planned that an additional 15 participants will be enrolled in Phase 1b of this study for additional experience with safety and efficacy, and to determine the recommended Phase 2 dose (RP2D) which may or may not be different from the MTD. Continued treatment will be available under this study protocol for up to eight 28-day cycles (Continued Treatment Phase) if the Investigator judges the benefit outweighs the risk. Once BTX-A51 treatment has completed, participants will be contacted by telephone every 3 months for up to 2 years after their last treatment for survival status and anticancer therapy (Overall Survival Follow-up).
    Location: Memorial Sloan Kettering Cancer Center, New York, New York

  • Reduced-Intensity Fludarabine, Melphalan, and Total Body Irradiation for the Treatment of Blood Cancer in Patients Undergoing Donor Stem Cell Transplant

    This phase II trial studies how well reduced-intensity fludarabine, melphalan, and total body irradiation work in treating patients with blood cancer who are undergoing donor stem cell transplant. Drugs used in chemotherapy, such as fludarabine and melphalan, 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. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving radiation therapy after chemotherapy may kill the remaining cancer cells.
    Location: Moffitt Cancer Center, Tampa, Florida

  • Venetoclax and Azacitidine for the Treatment of High-Risk Recurrent or Refractory Myelodysplastic Syndrome

    This phase I / II trial studies the side effects and best dose of venetoclax when given together with azacitidine in treating patients with high-risk myelodysplastic syndrome that has come back (recurrent) or does not respond to treatment (refractory). Drugs used in chemotherapy, such as venetoclax 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.
    Location: M D Anderson Cancer Center, Houston, Texas

  • Giving Chemotherapy and rATG for a Shortened Amount of Time before a Donor Stem Cell Transplantation for the Treatment of Patients with Blood Cancers

    This phase I trial studies the side effects of giving chemotherapy and a drug called rATG for a shorter period of time before a donor stem cell transplant in treating patients with blood cancers. This study will also look at whether the condensed regimen can shorten hospitalization following the transplantation. A chemotherapy regimen with the drugs busulfan, melphalan, and fludarabine may kill cancer cells in the body, making room in the bone marrow for new blood stem cells to grow and reducing the chance of transplanted cell rejection. The chemotherapy drugs work to interrupt the DNA (genetic information) in the cancer cells, stopping the cells from dividing and causing them to die. rATG targets and deactivates white blood cells called T cells that survive the chemotherapy. T cells may see the donor’s cells as foreign, causing a serious condition called graft-versus-host disease (GVHD). rATG helps prevent the donor stem cells from being rejected. Giving chemotherapy and rATG for a shorter period of time before a donor stem cell transplantation may help in reducing the number of side effects and shortening hospitalization following the transplantation.
    Location: Memorial Sloan Kettering Cancer Center, New York, New York

  • A Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

    A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following subjects: - Subjects receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the investigator, may derive clinical benefit in the opinion of the investigator from continuing treatment with luspatercept. - Placebo arm subjects from parent protocol (at the time of unblinding or in follow-up) crossing over to luspatercept treatment (provided subjects have met all requirements for entering the rollover study as per the parent protocol). - Subjects in the follow-up phase previously treated with luspatercept or placebo in the parent protocol will continue into long-term post-treatment follow-up in the rollover study until the follow-up commitments are met (unless they meet requirements as per parent protocol to cross-over to luspatercept treatment). The study design is divided into the Transition Phase, Treatment Phase and Follow-up Phase. Subjects will enter transition phase and depending on their background will enter either the treatment phase or the Long-term Post-treatment Follow-up (LTPTFU) phase. - Transition Phase (Screening): up to 21 days prior to enrollment - Treatment Phase: For subjects in luspatercept treatment the dose and schedule of luspatercept in this study will be the same as the last dose and schedule in the parent luspatercept study. For placebo arm subjects from parent protocol (at the time of unblinding or in follow-up) crossing over to luspatercept treatment (provided subjects have met all requirements for entering the rollover study as per the parent protocol) will start at a luspatercept dose of 1.0 mg / kg every 3 weeks (Q3W). This does not apply to subjects that are in long-term follow-up from the parent protocol. - Follow-up Phase: - 42 Day Safety Follow-up Phase: subjects will be followed for 42 days after the last dose of luspatercept, for the assessment of safety-related parameters and adverse event (AE) reporting. - Long-term Post-treatment Follow-up (LTPTFU) Phase: All subjects who are continuing in the LTPTFU Phase, will continue to be followed for 5 years from Dose 1 of the parent protocol, or 3 years of post-treatment from last dose of the parent protocol, whichever occurs later. Subjects will be followed every 6 months until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. Subjects will also be monitored for progression to AML or any malignancies / pre- malignancies. New anticancer or disease related therapies should be collected at the same time schedule. Subjects transitioning from a parent luspatercept study in post-treatment follow-up (safety or LTPTFU) will continue from the same equivalent point in this rollover study. The rollover study will be terminated, and relevant subjects will discontinue from the study when all subjects fulfill 5 years from Dose 1 of the parent protocol, or 3 years of post-treatment from last dose of the parent protocol, whichever occurs later. The shift to commercial drug is an alternative way to stop the study.
    Location: 2 locations

  • Ruxolitinib, Decitabine, and Donor Lymphocyte Infusion in Treating Patients with Relapsed Acute Myeloid Leukemia or Myelodysplastic Syndrome after Stem Cell Transplant

    This phase II trial studies how well ruxolitinib, decitabine, and donor white blood cells (donor lymphocyte infusion [DLI]) work in treating patients with acute myeloid leukemia or myelodysplastic syndrome that has come back after a stem cell transplant. Patients who have relapsed after a stem cell transplant commonly receive an infusion of immune cells from the original donor called a DLI. A DLI uses high dose chemotherapy prior to the infusion which increases the risk of graft versus host disease, a condition in which the transplanted cells attack the recipient’s body. While the cancer responds temporarily to high dose chemotherapy alone, it hasn’t been shown to bring about long-term remission. Instead of high dose chemotherapy, this study pairs DLI with decitabine, another chemotherapy drug, and adds ruxolitinib. Ruxolitinib is a type of drug called a "JAK" inhibitor and may help prevent graft-versus host disease. Giving ruxolitinib with decitabine and a DLI may decrease the risk of graft-versus host disease and increase the chances of remission.
    Location: 3 locations

  • PLX51107 and Azacitidine in Treating Patients with Acute Myeloid Leukemia or Myelodysplastic Syndrome

    This phase I trial studies the side effects and best dose of PLX51107 and how well it works with azacitidine in treating patients with acute myeloid leukemia or myelodysplastic syndrome. PLX51107 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as 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. Giving PLX51107 and azacitidine may work better than azacitidine alone in treating patients with acute myeloid leukemia or myelodysplastic syndrome.
    Location: M D Anderson Cancer Center, Houston, Texas

  • CYC065 CDK Inhibitor and Venetoclax Study in Relapsed / Refractory AML or MDS

    A Phase I Combination Study of CYC065 and Venetoclax for Relapsed or Refractory AML or MDS
    Location: M D Anderson Cancer Center, Houston, Texas

  • ASTX727 and FT-2102 in Treating Patients with IDH1-Mutated Recurrent or Refractory Myelodysplastic Syndrome or Acute Myeloid Leukemia

    This phase Ib / II trial studies the side effects and best dose of FT-2102 when given together with ASTX727 in treating patients with IDH1-mutated myelodysplastic syndrome or acute myeloid leukemia that has come back (recurrent) or does not respond to treatment (refractory). ASTX727 is an oral deoxyribonucleic acid (DNA) methyltransferase (DNMT) inhibitor. DNA methylation is necessary for cell differentiation and development. Changes to the methylation profile can lead to DNA instability which can cause diseases like cancer. DNMT inhibitors target and inhibit these changes. FT-2102 is an isocitrate dehydrogenase 1 (IDH1) inhibitor. IDH1 is a type of protein involved in metabolism, or the process of providing the body’s cells with energy. FT-2102 may stop the abnormal IDH1 protein and may reduce 2-HG levels in diseased cells to levels found in normal cells. Giving ASTX727 and FT-2102 may work better in treating patients with myelodysplastic syndrome or acute myeloid leukemia compared to ASTX727 and FT-2102 alone.
    Location: Vanderbilt University / Ingram Cancer Center, Nashville, Tennessee

  • CPX-351 in Treating Patients with Myelodysplastic Syndromes after Hypomethylating Agent Failure

    This phase II trial studies how well CPX-351 works in treating patients with myelodysplastic syndromes, who have failed previous treatment with azacitidine or decitabine (hypomethylating agents). CPX-351 is an investigational (experimental) drug for the treatment of myelodysplastic syndrome that works by delivering two chemotherapy medications (daunorubicin and cytarabine) together which are then concentrated into the bone marrow (the part of the body that makes blood cells). Drugs used in chemotherapy, such as daunorubicin 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: Case Comprehensive Cancer Center, Cleveland, Ohio

  • ONC 201 Maintenance Therapy for the Treatment of Patients with Acute Myeloid Leukemia or Myelodysplastic Syndrome after Donor Stem Cell Transplant

    This phase I trial studies the side effects of ONC 201 as maintenance therapy in treating patients with acute myeloid leukemia or myelodysplastic syndrome after receiving a donor stem cell transplant. ONC 201 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: University of Nebraska Medical Center, Omaha, Nebraska

  • Adoptive Cellular Therapy (PRGN-3006 T Cells) in Treating Patients with Recurrent or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

    This phase I / Ib trial studies the side effects and best dose of PRGN-3006 T cells in treating patients with acute myeloid leukemia that has come back or does not respond to treatment or high-risk myelodysplastic syndrome. PRGN-3006 T cells uses the patient’s own genetically altered blood cells to treat cancer. PRGN-3006 T cells are made from white blood cells that are removed from the patient. The cells are genetically modified to create a protein (called a chimeric antigen receptor or CAR) on the surface of T cells, a type of blood cell that fights infection and cancer cells. The hope is that the CAR on the T cells will bind to and kill cells that express CD33, a molecule that is found on the surface of cancer cells from myelodysplastic syndrome or acute myeloid leukemia patients.
    Location: Moffitt Cancer Center, Tampa, Florida

  • Valproic Acid Expanded Umbilical Cord Blood Stem Cells in Treating Adult Patients with Hematological Malignancies Undergoing Donor Stem Cell Transplant

    This phase I trial studies the effects of valproic acid expanded umbilical cord blood stem cells in treating adult patients with hematological malignancies undergoing donor stem cell transplant. Expanding or growing umbilical cord blood stem cells in a laboratory using valproic acid may lead to faster white blood cell count recovery, lower the risk of infections, and improve transplant results compared to umbilical cord blood stem cells that have not been expanded.
    Location: Icahn School of Medicine at Mount Sinai, New York, New York

  • A Phase I Study of CYC140, a PLK-1 Inhibitor, in Advanced Leukemias or MDS

    A Phase I Pharmacologic Study of CYC140, a polo-like kinase 1 inhibitor, in Patients with Advanced Leukemias or Myelodysplastic Syndromes
    Location: M D Anderson Cancer Center, Houston, Texas

  • Azacitidine, Venetoclax, and Pevonedistat in Treating Patients with Newly Diagnosed Acute Myeloid Leukemia

    This phase I / II trial studies the best dose of venetoclax when given together with azacitidine and pevonedistat and to see how well it works in treating patients with newly diagnosed acute myeloid leukemia. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Venetoclax and pevonedistat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving azacitidine, venetoclax, and pevonedistat may work better in treating patients with acute myeloid leukemia.
    Location: M D Anderson Cancer Center, Houston, Texas

  • Stem Cell Transplant with Chemotherapy and Selected Use of Blinatumomab in Treating Patients with Blood Cancer

    This phase II trial studies how well transplanting blood cells with chemotherapy work in treating patients with a high risk blood cancer that is in remission. Giving chemotherapy before a donor stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells and cancer cells. It may also help 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 (called graft versus host disease). Giving filgrastim may stop this from happening. Immunotherapy with monoclonal antibodies, such as blinatumomab, may induce changes in the body's immune system and may interfere with the ability of cancer cells to grow and spread. Giving stem cells with chemotherapy and blinatumomab may work better in treating patients with blood cancer.
    Location: St. Jude Children's Research Hospital, Memphis, Tennessee

  • CD45RA Depleted T Cell Infusion after Alpha / Beta Depleted Stem Cell Transplant for the Reduction of Complications in Patients at Risk for Viral or Fungal Infections

    This phase I trial studies the side effects of CD45RA depleted T cell infusion given after an alpha / beta depleted stem cell transplant, and to see how well it works in reducing complications in patients at risk for viral or fungal infections. There is a higher rate of complications using cells from an unrelated or partially matched related donor. Some donor cells may cause a complication called graft versus host disease (GVHD) and an infection with the Epstein-Barr virus (EBV). In this study, the CliniMACs device is used before the transplant to remove alpha / beta T cells that may cause GVHD and B cells that carry EBV. The device is also used to remove CD45RA T cells. Giving CD45RA depleted T cells after the transplant may reduce some of the complications of the transplant and decrease the time it takes for the new stem cells to grow in the body.
    Location: Children's Hospital of Philadelphia, Philadelphia, Pennsylvania

  • Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults with Blood Cancers Undergoing Donor Stem Cell Transplant

    This phase II trial studies how well naive T-cell depletion works in preventing chronic graft-versus-host disease in children and young adults with blood cancers undergoing donor stem cell transplant. Sometimes the transplanted white blood cells from a donor attack the body’s normal tissues (called graft versus host disease). Removing a particular type of T cell (naive T cells) from the donor cells before the transplant may stop this from happening.
    Location: 7 locations

  • A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)

    The goal of this clinical research study is to determine whether it is safe and practical to give CK0801 (a Cord blood-derived T-regulatory cell product) to patients with bone marrow failure syndrome. Researchers want to determine the highest possible dose that is safe to be given. Researchers also want to learn if CK0801 may improve the symptoms of bone marrow failure syndrome. Patients enrolled in this study will all have been diagnosed with treatment refractory bone marrow failure syndrome (which includes aplastic anemia, myelodysplastic syndrome, or myelofibrosis). Participants eligible to participate in this study are unable or unwilling to be treated with standard therapy or have failed standard therapy.
    Location: M D Anderson Cancer Center, Houston, Texas


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