Clinical Trials Using Azacitidine

Clinical trials are research studies that involve people. The clinical trials on this list are studying Azacitidine. 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 71
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  • Azacitidine and Combination Chemotherapy in Treating Infants with Acute Lymphoblastic Leukemia and KMT2A Gene Rearrangement

    This pilot phase II trial studies the side effects of azacitidine and combination chemotherapy in infants with acute lymphoblastic leukemia and KMT2A gene rearrangement. Drugs used in chemotherapy, such as methotrexate, prednisolone, daunorubicin hydrochloride, cytarabine, dexamethasone, vincristine sulfate, pegaspargase, hydrocortisone sodium succinate, azacitidine, cyclophosphamide, mercaptopurine, leucovorin calcium, and thioguanine 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 may kill more cancer cells.
    Location: 158 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

  • 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 the relevant Phase 1 cohorts, Phase 2 will begin enrollment. Patients will be enrolled across 8 different cohorts, examining the effect of FT-2102 (as a single agent) and FT-2102 + azacitidine (combination) on various AML / MDS disease states.
    Location: 15 locations

  • Pevonedistat, Azacitidine, Fludarabine Phosphate, and Cytarabine in Treating Patients with Relapsed or Refractory Acute Myeloid Leukemia or Relapsed High-Risk 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 that has come back or has not responded to treatment or high-risk myelodysplastic syndrome that has come back. Pevonedistat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as azacitidine, and 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: 14 locations

  • APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS)

    A Phase III, multicenter, randomized study to compare the rate of complete response (CR) and duration of CR, in patients with TP53-mutated MDS who will receive APR-246 and azacitidine or azacitidine alone.
    Location: 13 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: 13 locations

  • A Study of FT 2102 in Participants With Advanced Solid Tumors and Gliomas With an IDH1 Mutation

    This Phase 1 / 2 study will evaluate the safety, efficacy, PK, and PD of FT-2102 as a single agent and in combination with other anti-cancer drugs in patients with advanced solid tumors and gliomas. The study is divided into two parts: single agent FT-2102 followed by combination therapy. Part 1: A single agent, open-label study in up to five cohorts (glioma, hepatobiliary tumors, chondrosarcoma, intrahepatic cholangiocarcinoma, and other IDH1 mutant solid tumors) that will include a Phase 1 dose confirmation followed by a Phase 2 investigation of clinical activity in up to 4 cohorts. During the dose confirmation, additional doses or altered dose schedules may be explored. Part 2: An open-label study of FT-2102 in combination with other anti-cancer agents. Patients will be enrolled across 4 different disease cohorts, examining the effect of FT-2102 + azacitidine (glioma and chondrosarcoma), FT-2102+nivolumab (hepatobiliary tumors) and FT-2102+gemcitabine / cisplatin (intrahepatic cholangiocarcinoma). There will be a safety lead-in followed by a Phase 2 evaluation in up to four cohorts of patients.
    Location: 13 locations

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

    This is a Phase 1b, 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: 10 locations

  • An Open-Label, Dose-Escalation / Dose-Expansion Safety Study of INCB059872 in Subjects With Advanced Malignancies

    This is an open-label, dose-escalation / dose-expansion study of INCB059872 in subjects with advanced malignancies. The study will be conducted in 4 parts. Part 1 (mono therapy dose escalation) will determine the recommended dose(s) of INCB059872 for dose expansion, based on maximum tolerated dose and / or a tolerated pharmacologically active dose. Part 2 (dose expansion) will further determine the safety, tolerability, efficacy, PK, and PD of the selected monotherapy dose(s) in AML / MDS, SCLC, myelofibrosis, Ewing sarcoma, and poorly differentiated neuroendocrine tumors. Part 3 will determine the recommended dose(s) of INCB059872 in combination with azacitadine and all-trans retinoic acid in AML and in combination with nivolumab in SCLC. Part 4 will further determine the safety, tolerability, efficacy, PK, and PD of the selected combination dose(s) in Part 3.
    Location: 10 locations

  • Study of INCB053914 in Subjects With Advanced Malignancies

    This is an open-label, dose-escalation study of the proviral integration site of Moloney murine leukemia virus (PIM) kinase inhibitor INCB053914 in subjects with advanced malignancies. The study will be conducted in 4 parts. Part 1 (monotherapy dose escalation) will evaluate safety and determine the maximum tolerated dose of INCB053914 monotherapy and the recommended phase 2 dose(s) (a tolerated pharmacologically active dose that will be taken forward into the remaining parts of the study). Part 2 (monotherapy dose expansion) will further evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of the recommended Phase 2 dose(s). Part 3 (combination dose finding) will evaluate safety of INCB053914 in combination with select standard of care (SOC) agents and will identify the optimal INCB053914 dose in combination with conventional SOC regimens to take forward into Part 4. Part 4 (combination dose expansion) will further evaluate the safety, efficacy and pharmacokinetics of the recommended Phase 2 dose combination(s).
    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: 9 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 (SY-1425 administered as a monotherapy or in combination with azacitidine), 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).
    Location: 9 locations

  • A Study Evaluating Intensive Chemotherapy With or Without Glasdegib or Azacitidine With or Without Glasdegib In Patients With Previously Untreated Acute Myeloid Leukemia

    Glasdegib is being studied in combination with azacitidine for the treatment of adult patients with previously untreated acute myeloid leukemia (AML) who are not candidates for intensive induction chemotherapy (Non-intensive AML population). Glasdegib is being studied in combination with cytarabine and daunorubicin for the treatment of adult patients with previously untreated acute myeloid leukemia (Intensive AML population).
    Location: 10 locations

  • 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)

    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).
    Location: 7 locations

  • A Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients With Relapsed or Refractory Malignancies

    An open-label, global, multi-center study to evaluate the safety and pharmacokinetics of venetoclax monotherapy, to determine the dose limiting toxicity (DLT) and the recommended Phase 2 dose (RPTD), and to assess the preliminary efficacy of venetoclax in pediatric and young adult participants with relapsed or refractory malignancies.
    Location: 8 locations

  • An Efficacy and Safety Study Of Pracinostat In Combination With Azacitidine In Adults With Acute Myeloid Leukemia

    This is a Phase III, multicenter, double-blind, randomized study of pracinostat vs. placebo with azacitidine (AZA) as background therapy in patients ≥ 18 years of age with newly diagnosed acute myeloid leukemia (AML), excluding acute promyelocytic leukemia and cytogenetic low-risk AML, who are unfit to receive intensive remission induction chemotherapy due to age ≥ 75 years or comorbidities. Patients will be randomized in a 1:1 ratio to one of two groups: Group A (experimental group) to receive pracinostat plus AZA and Group B (control group) to receive placebo plus AZA. Randomization will be stratified by cytogenetic risk category (intermediate vs. unfavorable-risk, according to SWOG Cytogenetic Risk Category Definitions) and ECOG performance status (0-1 vs. 2). Treatments will be administered based on 28-day cycles, with pracinostat / placebo administered orally once every other day, 3 times a week for 3 weeks, followed by one week of no treatment and AZA administered for 7 days of each cycle. Study treatment should continue until there is documented disease progression, relapse from complete remission (CR), or non-manageable toxicity. A minimum of 6 cycles may be required to achieve a complete remission. Once permanently discontinued from study treatment, patients will enter the Long-term Follow-up phase of the study and will be followed for assessment of disease progression, if applicable, and survival every 3 months (±1 month) until death. The end of this study is defined when 390 events (deaths) have occurred and the study is unblinded for final overall survival analysis. Patients who are receiving study treatment at the end of the study may have the opportunity to continue to receive the study drugs to which they were randomized to (Post- Study Observation Period), until the Sponsor informs the Investigators of the appropriate course of action based on the study results. The Post-Study Observation Period is defined as the period starting from the end of the study for a maximum of 12 months.
    Location: 7 locations

  • Azacitidine, Entinostat, and Nivolumab or Nivolumab Alone in Treating Patients with Recurrent Metastatic Non-small Cell Lung Cancer

    This phase II trial studies how well azacitidine, entinostat, and nivolumab or nivolumab alone work in treating patients with non-small cell lung cancer that has come back and has spread to other places in the body. 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. Entinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Monoclonal antibodies, such as nivolumab, may kill tumor cells that are left after chemotherapy. It is not yet known if azacitidine and entinostat followed by nivolumab or nivolumab alone is more effective in treating non-small cell lung cancer.
    Location: 7 locations

  • Hu5F9-G4 Monotherapy or Hu5F9-G4 in Combination With Azacitidine in Patients With Hematological Malignancies

    This trial will evaluate Hu5F9-G4, a monoclonal antibody which is designed to block a protein called CD47, which is widely expressed on human cancer cells. Blocking CD47 with Hu5F9-G4 may enable the body's immune system to find and destroy the cancer cells. In this study, Hu5F9-G4 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 Hu5F9-G4 monotherapy in a relapsed / refractory AML and MDS population, and of Hu5F9-G4 in combination with azacitidine in previously untreated AML and MDS; and to evaluate the efficacy of Hu5F9-G4 monotherapy in relapsed / refractory AML / MDS, and of Hu5F9-G4 in combination with azacitidine in previously untreated AML / MDS, as measured by the objective response rate.
    Location: 6 locations

  • PRIMA-1 Analog APR-246 and Azacitidine in Treating Patients with TP53 Mutant Myeloid Cancers

    This phase Ib / II trial studies the side effects and best dose of PRIMA-1 analog APR-246 when given together with azacitidine and to see how well they work in treating patients with TP53 mutant myeloid cancers. Giving PRIMA-1 analog APR-246 and azacitidine may work better in treating patients with TP53 mutant myeloid cancers.
    Location: 6 locations

  • A Study of ASP2215 (Gilteritinib) by Itself, ASP2215 Combined With Azacitidine or Azacitidine by Itself to Treat Adult Patients Who Have Recently Been Diagnosed With Acute Myeloid Leukemia With a FLT3 Gene Mutation and Who Cannot Receive Standard Chemotherapy

    This is a clinical study for adult patients who have recently been diagnosed with acute myeloid leukemia or AML. AML is a type of cancer. It is when bone marrow makes white blood cells that are not normal. These are called leukemia cells. Some patients with AML have a mutation, or change, in the FLT3 gene. This gene helps leukemia cells make a protein called FLT3. This protein causes the leukemia cells to grow faster. For patients with AML who cannot receive standard chemotherapy, azacitidine (also known as Vidaza®) is a current standard of care treatment option in the United States. This clinical study is testing an experimental medicine called ASP2215, also known as gilteritinib. Gilteritinib works by stopping the leukemia cells from making the FLT3 protein. This can help stop the leukemia cells from growing faster. This study will compare two different treatments. Patients are assigned to one of these two groups by chance: a medicine called azacitidine, also known as Vidaza®, or an experimental medicine gilteritinib in combination with azacitidine. There is a twice as much chance to receive both medicines combined than azacitidine alone. The clinical study may help show which treatment helps patients live longer.
    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

  • 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 have fails primary therapy and that does not respond to treatment or has come back. Pevonedistat and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: 5 locations

  • A Study of DCLL9718S in Participants With Relapsed or Refractory Acute Myeloid Leukemia (AML) or DCLL9718S in Combination With Azacitidine in Participants With Previously Untreated AML Unsuitable for Intensive Induction Chemotherapy

    This Phase Ia / Ib, open-label, multicenter study will evaluate the safety, tolerability, and preliminary efficacy of DCLL9718S as a single agent (Phase Ia, Arm A) in participants with relapsed or refractory AML or in combination with azacitidine (Phase Ib, Arm B) in participants with previously untreated AML who are not eligible for intensive induction chemotherapy. Each arm will consist of two stages: a dose-escalation stage and an expansion stage. The dose-escalation stage is designed to establish the maximum tolerated dose (MTD) and recommended Phase II dose (RP2D) for DCLL9718S alone (Arm A) or in combination with azacitidine (Arm B). The dose-expansion stage is designed to characterize the long-term safety and tolerability of DCLL9718S.
    Location: 4 locations

  • Tagraxofusp-erzs and Azacitidine in Treating Patients with Myelodysplastic Syndrome or Acute Myeloid Leukemia That Is Untreated, Relapsed, or Refractory

    This phase I trial studies the side effects and best dose of tagraxofusp-erzs (SL-401) when given together with azacitidine in treating patients with myelodysplastic syndrome or acute myeloid leukemia that is untreated, has come back, or does not respond to treatment. Combinations of biological substances in tagraxofusp-erzs may be able to carry cancer-killing substances directly to cancer cells. 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 tagraxofusp-erzs and azacitidine may work better in treating patients with myelodysplastic syndrome or acute myeloid leukemia.
    Location: 4 locations


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