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Myelodysplastic Syndrome clinical trials at UC Health
36 in progress, 19 open to new patients

  • A Combination Study of PF-04449913 (Glasdegib) and Azacitidine In Untreated MDS, AML and CMML Patients

    open to eligible people ages 18 years and up

    This multi center open label Phase 1b study is designed to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of glasdegib (PF-04449913) when combined with azacitidine in patients with previously untreated Higher Risk Myelodysplastic Syndrome (MDS), Acute Myeloid Leukemia (AML), or Chronic Myelomonocytic Leukemia (CMML). This clinical study includes two components: (a) a safety lead in cohort (LIC) and (b) an expansion phase with an AML cohort and an MDS cohort.

    at UCSD

  • A Phase 2 Study of CPI-0610 With and Without Ruxolitinib in Patients With Myelofibrosis

    open to eligible people ages 18 years and up

    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.

    at UCLA

  • A Study of Experimental FT-2102 in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome (types of blood system cancer)

    “This study will evaluate the safety, efficacy, PK, and PD of FT-2102 as a single agent or in combination with azacitidine or cytarabine.”

    open to eligible people ages 18 years and up

    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.

    at UCLA UC Davis

  • A Study of Experimental Treatment With Ibrutinib and Lenalidomide in Patients With Myelodysplastic Syndrome

    “This is a study of combined medication treatment for patients with Myelodysplastic Syndrome.”

    open to eligible people ages 18 years and up

    This phase I trial studies the side effects and best dose of ibrutinib when giving together with lenalidomide in treating patients with myelodysplastic syndrome. Ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as lenalidomide, 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 ibrutinib and lenalidomide may work better in treating patients with myelodysplastic syndrome.

    at UC Davis

  • Collection of Samples From Patients With MDS

    open to eligible people ages 18 years and up

    The purpose of this study is to collect information and bone marrow, blood, saliva, cheek cells and skin to be used in the laboratory to assist the sponsor in identifying a new way of treating MDS.

    at UCSD

  • Connect® MDS/AML Disease Registry

    open to eligible people ages 18 years and up

    The purpose of the Connect® MDS/AML Disease Registry is to provide unique insights into treatment regimens and sequencing of these regimens as they relate to clinical outcomes of patients with newly diagnosed MDS, ICUS or AML in routine clinical practice and evaluate molecular and cellular markers that may provide further prognostic classification and/or might be predictive of therapy outcomes.

    at UCSD

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

    open to eligible people ages 18-81

    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.

    at UCLA UCSD

  • Dose Escalation of OXi4503 as Single Agent and Combination With Cytarabine w/Subsequent Ph 2 Cohorts for AML and MDS

    open to eligible people ages 18 years and up

    Phase 1 will investigate maximum tolerated dose of OXi4503 as a single agent and in combination with intermediate-dose cytarabine in subjects with relapsed/refractory AML or MDS. Phase 2 will investigate overall response rate of OXi4503 in combination with intermediate-dose cytarabine in 1) subjects with MDS after failure of 1 prior hypomethylating agent (Arm A) and 2) subjects with relapsed and refractory AML after treatment failure of up to 1 prior chemotherapy regimen (Arm B).

    at UCLA

  • Ibrutinib and Azacitidine for Treatment of Higher Risk Myelodysplastic Syndrome

    open to eligible people ages 18 years and up

    This phase Ib trial studies the side effects and best dose of ibrutinib when given together with azacitidine in treating patients with myelodysplastic syndrome that is likely to occur or spread (higher risk) and who were previously treated or untreated and unfit for or refused intense therapy. Ibrutinib and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

    at UCSF UC Davis UCLA UC Irvine UCSD

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

    open to eligible people ages 18 years and up

    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.

    at UC Davis

  • Ipilimumab or Nivolumab in Treating Patients With Relapsed Hematologic Malignancies After Donor Stem Cell Transplant

    open to eligible people ages 18 years and up

    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.

    at UCSD UCLA

  • Personalized Adoptive Cellular Therapy Targeting MDS Stem Cell Neoantigens (PACTN)

    open to eligible people ages 18 years and up

    This study will evaluate the safety of autologous T cells that have been immunized ex vivo with patient-specific MDS stem cell neoantigens in patients with MDS.

    at UCSD

  • Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML), or Low-Blast Acute Myelogenous Leukemia (AML)

    open to eligible people ages 18 years and up

    The purpose of this study is to determine whether the combination of pevonedistat and azacitidine improves event-free survival (EFS) when compared with single-agent azacitidine (An event is defined as death or transformation to AML in participants with MDS or CMML, whichever occurs first, and is defined as death in participants with low-blast AML).

    at UCSD

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

    open to eligible people ages 18-99

    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

    at UCLA

  • Response-Based Chemotherapy to treat newly diagnosed Acute Myeloid Leukemia /Myelodysplastic Syndrome patients with Down syndrome

    “Response-based chemotherapy separates patients into different risk groups according to how they respond to the first course of treatment”

    open to eligible people ages up to 3 years

    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.

    at UCSF UC Davis UCLA

  • Study of Lenalidomide in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome

    open to eligible people ages 18-65

    The purpose of this study is to determine whether lenalidomide can stop the growth of leukemia stem cells and can be used to prevent the return of leukemia cells after a transplant.

    at UC Davis

  • Study of ProTmune for Allogeneic HCT in Adult Patients With Hematologic Malignancies

    open to eligible people ages 18-70

    This study is a Phase 1, Non-randomized, Open-label/Phase 2 Randomized, Blinded Study of ProTmune (ex vivo programmed mobilized peripheral blood cells) Versus Non-programmed mobilized peripheral blood cells for Allogeneic Hematopoietic Cell Transplantation (HCT) in Adult Subjects Aged 18 to 70 years with Hematologic Malignancies. A maximum of 70 total eligible subjects will be enrolled and treated in the trial at approximately 15-20 centers in the US and EU.

    at UCSD

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

    open to eligible people ages 18 years and up

    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.

    at UCLA

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

    open to eligible people ages 12-65

    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.

    at UCLA

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

    Sorry, currently not accepting new patients, but might later

    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.

    at UCSF

  • A Study To Evaluate PF-04449913 With Chemotherapy In Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

    Sorry, in progress, not accepting new patients

    This is a study to evaluate PF-04449913 (an inhibitor of the Hedgehog pathway) in Acute Myeloid Leukemia and high-risk Myelodysplastic Syndrome in combination with standard agents used to treat these diseases.

    at UCSD UCLA

  • An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML)

    Sorry, in progress, not accepting new patients

    The purpose of this study is to evaluate the efficacy and safety of pevonedistat plus azacitidine versus single-agent azacitidine in participants with HR-MDS or CMML, or low-blast AML.

    at UCSD

  • 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

    Sorry, currently not accepting new patients, but might later

    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. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor 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.

    at UC Davis

  • Collecting and Storing Blood, Bone Marrow, and Other Samples From Patients With Acute Leukemia, Chronic Leukemia, or Myelodysplastic Syndromes

    Sorry, in progress, not accepting new patients

    As one of the nation's largest cooperative cancer treatment groups, the Alliance for Clinical Trials in Oncology (Alliance) is in a unique position to organize a Leukemia Tissue Bank. The member institutions diagnose hundreds of patients with leukemia or myelodysplastic syndrome each year, and uniformly treat these patients with chemotherapy regimens. The Alliance offers centralized data management for the clinical history, the classification of the leukemia and myelodysplastic syndrome, cytogenetics, flow cytometric analysis, treatment and follow-up. The highly skilled health care providers at each member institution are familiar with obtaining informed consent, completing data questionnaires and shipping specimens. There currently exists a central processing facility where samples are prepared for a variety of cellular and molecular studies. Hence, the patient resources, the health care providers, and a processing facility for a Leukemia Tissue Bank are all in place. What is needed, however, and is addressed in the current protocol, is a formal mechanism to procure bone marrow, blood and normal tissue from patients with hematologic malignancies who are to be enrolled on Alliance (Cancer and Leukemia Group B [CALGB]) treatment studies.

    at UCSF

  • Cytogenetic Studies in Acute Leukemia and Multiple Myeloma

    Sorry, in progress, not accepting new patients

    Chromosomal analysis or the study of genetic differences in patients previously untreated with AML, ALL, MDS or MM may be helpful in the diagnosis and classification of disease. It may also improve the ability to predict the course of disease and the selection of therapy. Institutions must have either an Alliance-approved cytogeneticist or an agreement from an Alliance-approved main member cytogenetics laboratory to enroll a patient on CALGB 8461. The Alliance Approved Institutional Cytogeneticists list is posted on the Alliance for Clinical Trials in Oncology website.

    at UCSF UCSD

  • Donor Lymphocyte Infusion With Azacitidine to Prevent Hematologic Malignancy Relapse After Stem Cell Transplantation

    Sorry, in progress, not accepting new patients

    The goal of this study is to determine whether post-transplant consolidation with azacitidine combined with donor lymphocyte infusion (DLI) is a safe and effective approach for the prevention of relapse in pediatric and young adult patients with hematologic malignancies who have undergone hematopoietic stem cell transplantation (HSCT).

    at UCSF

  • Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

    Sorry, not accepting new patients

    This protocol provides expanded access to bone marrow transplants for children who lack a histocompatible (tissue matched) stem cell or bone marrow donor when an alternative donor (unrelated donor or half-matched related donor) is available to donate. In this procedure, some of the blood forming cells (the stem cells) are collected from the blood of a partially human leukocyte antigen (HLA) matched (haploidentical) donor and are transplanted into the patient (the recipient) after administration of a "conditioning regimen". A conditioning regimen consists of chemotherapy and sometimes radiation to the entire body (total body irradiation, or TBI), which is meant to destroy the cancer cells and suppress the recipient's immune system to allow the transplanted cells to take (grow). A major problem after a transplant from an alternative donor is increased risk of Graft-versus-Host Disease (GVHD), which occurs when donor T cells (white blood cells that are involved with the body's immune response) attack other tissues or organs like the skin, liver and intestines of the transplant recipient. In this study, stem cells that are obtained from a partially-matched donor will be highly purified using the investigational CliniMACS® stem cell selection device in an effort to achieve specific T cell target values. The primary aim of the study is to help improve overall survival with haploidentical stem cell transplant in a high risk patient population by limiting the complication of GVHD.

    at UCSF

  • Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia

    Sorry, in progress, not accepting new patients

    This phase II trial studies how well total-body irradiation (TBI) works when given together with fludarabine phosphate and cyclophosphamide followed by donor bone marrow transplant, mycophenolate mofetil, and cyclosporine in treating patients with Fanconi anemia (FA). Giving low doses of chemotherapy, such as fludarabine phosphate and cyclophosphamide, and TBI before or after a donor bone marrow transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving mycophenolate mofetil and cyclosporine after the transplant may stop this from happening.

    at UCSF

  • KIR Favorable Mismatched Haplo Transplant and KIR Polymorphism in ALL/AML/MDS Allo-HCT Children

    Sorry, accepting new patients by invitation only

    This is a phase II, open-label, non-randomized, prospective study of haploidentical transplantation using KIR-favorable donors for children with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT). The relationship of KIR2DL1 polymorphisms to survival in children with these diseases undergoing any approach to allogeneic HCT during the study time frame will also be determined.

    at UCSF

  • Lenalidomide With or Without Epoetin Alfa in Treating Patients With Myelodysplastic Syndrome and Anemia

    Sorry, in progress, not accepting new patients

    This randomized phase III trial studies lenalidomide to see how well it works with or without epoetin alfa in treating patients with myelodysplastic syndrome and anemia. Lenalidomide may stop the growth of myelodysplastic syndrome by blocking blood flow to the cells. Colony stimulating factors, such as epoetin alfa, may increase the number of immune cells found in bone marrow or peripheral blood. It is not yet known whether lenalidomide is more effective with or without epoetin alfa in treating patients with myelodysplastic syndrome and anemia.

    at UC Davis

  • Phase I Dose Finding and Proof-of-concept Study of Panobinostat With Standard Dose Cytarabine and Daunorubicin for Untreated Acute Myeloid Leukemia or Advanced Myelodysplastic Syndrome

    Sorry, in progress, not accepting new patients

    The purpose of this study is to see if Panobinostat is safe to give to patients and to determine the best dose to give in combination with standard cytarabine and daunorubicin chemotherapy.

    at UCSF

  • Randomized Study of ON 01910.Na in Refractory Myelodysplastic Syndrome Patients With Excess Blasts

    Sorry, in progress, not accepting new patients

    The primary objective of this study is to compare overall survival (OS) in patients receiving ON 01910.Na + best supportive care (BSC) to OS of patients receiving BSC in a population of patients with myelodysplastic syndrome (MDS) with excess blasts (5% to 30% bone marrow blasts) who have failed azacitidine or decitabine treatment. This patient population has no available therapy and a short life expectancy (approximately 4 months). The high level of bone marrow activity of ON 01910.Na documented in Phase 1 and 2 studies has the potential to delay substantially the transition of MDS to Acute Myeloid Leukemia(AML), a very significant and severe complication, which shortens survival of these MDS patients.

    at UCSD

  • Safety Study of AG-120 or AG-221 in Combination With Induction and Consolidation Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia With an IDH1 and/or IDH2 Mutation

    Sorry, in progress, not accepting new patients

    The purpose of this Phase I, multicenter, clinical trial is to evaluate the safety of AG-120 and AG-221 when given in combination with standard AML induction and consolidation therapy. The study plans to evaluate 1 dose level of AG-120 in subjects with an IDH1 mutation and 1 dose level (and 2 dose schedules) of AG-221 in subjects with an IDH2 mutation. AG-120 or AG-221 will be administered with 2 types of AML induction therapies (cytarabine with either daunorubicin or idarubicin) and 2 types of AML consolidation therapies (mitoxantrone with etoposide [ME] or cytarabine). After consolidation therapy, subjects may continue on to maintenance therapy and receive daily treatment of AG-120 or AG-221 until the end of study or until relapse, development of an unacceptable toxicity, or hematopoietic stem cell transplant (HSCT). End of study is defined as 2 years from the time the last subject enrolls, or sooner, if all subjects have come off study (or withdrew consent) prior to 2 years after the last subject has enrolled.

    at UCLA

  • Safety, Tolerability and Pharmacokinetics of Milademetan Alone and With 5-Azacitidine (AZA) in Acute Myelogenous Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS)

    Sorry, not currently recruiting here

    This study will take place in parts: - Dose Escalation (Part 1): Participants receive milademetan alone with different dose schedules - Dose Escalation (Part 1A): Participants receive milademetan in combination with AZA, with different dose schedules The recommended dose for Part 2 will be selected. - Dose Expansion (Part 2): After Part 1A, participants will receive the recommended Part 2 dose schedule. There will be three groups - those with: 1. refractory or relapsed AML 2. newly diagnosed AML unfit for intensive chemotherapy 3. high-risk MDS - End-of-Study Follow-Up: Safety information will be collected until 30 days after the last treatment. This is the end of the study. The recommended dose for the next study will be selected.

    at UCSF

  • 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)

    Sorry, in progress, not accepting new patients

    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).

    at UCSD

  • Vosaroxin and Infusional Cytarabine in Treating Patients With Untreated Acute Myeloid Leukemia

    Sorry, currently not accepting new patients, but might later

    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.

    at UCLA

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