Clinical Trials Using IDH1 Inhibitor AG-120

Clinical trials are research studies that involve people. The clinical trials on this list are studying IDH1 Inhibitor AG-120. 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 1-11 of 11
  • Ivosidenib in Treating Patients with Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders with IDH1 Mutations (A Pediatric MATCH Treatment Trial)

    This phase II Pediatric MATCH trial studies how well ivosidenib works in treating patients with solid tumors that have spread to other places in the body (advanced), lymphoma, or histiocytic disorders that have IDH1 genetic alterations (mutations). Ivosidenib may block the growth of cancer cells that have specific genetic changes in an important signaling pathway called the IDH pathway.
    Location: 168 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

  • Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation

    The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced hematologic malignancies that harbor an IDH1 mutation. The first portion of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of AG-120 to determine maximum tolerated dose (MTD) and / or the recommended Phase II dose. The second portion of the study is a dose expansion phase where four cohorts of patients will receive AG-120 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose. Additionally, the study includes a substudy evaluating the safety and tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of AG-120 in subjects with relapsed or refractory myelodysplastic syndrome with an IDH1 mutation. Anticipated time on study treatment is until disease progression or unacceptable toxicity occurs.
    Location: 9 locations

  • Ivosidenib as Maintenance Therapy in Treating Patients with IDH1-mutant Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia following Stem Cell Transplant

    This phase I trial studies the best dose and side effects of ivosidenib as maintenance therapy in treating patients with IDH1-mutant acute myeloid leukemia, myelodysplastic syndrome, or chronic myelomonocytic leukemia following stem cell transplant. Ivosidenib is an inhibitor of the protein IDH1. IDH1 is an enzyme that, when mutated, can overproduce metabolites (substances that help with metabolism) and compounds that contribute to the growth of cancer cells. Ivosidenib may help block the over production of these substances and possibly reduce the chances of relapse in patients with IDH1-mutant myeloid cancers.
    Location: 4 locations

  • Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Patients With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation

    Study AG120-C-009 is a global, Phase 3, multicenter, double-blind, randomized, placebo-controlled clinical trial to evaluate the efficacy and safety of AG-120 (ivosidenib) + azacitidine vs placebo + azacitidine in adult participants with previously untreated IDH1m AML who are considered appropriate candidates for non-intensive therapy. The primary endpoint is event-free survival (EFS). The key secondary efficacy endpoints are overall survival (OS), rate of complete remission (CR), rate of CR and complete remission with partial hematologic recovery (CRh), and overall response rate (ORR). Participants eligible for study treatment based on Screening assessments will be randomized 1:1 to receive oral AG-120 or matched placebo, both administered in combination with subcutaneous (SC) or intravenous (IV) azacitidine. An estimated 200 participants will take part in the study.
    Location: 2 locations

  • AG-120 in People with IDH1 Mutant Chondrosarcoma

    This phase II trial studies how well AG-120 works in treating patients with IDH1 mutant chondrosarcoma that has spread to nearby tissue or lymph nodes (locally advanced), spread to other places in the body (metastatic), or has come back (recurrent). IDH1 is a type of protein involved in the process of providing energy to the body’s cells. In diseases like sarcoma, an abnormal form (mutated form) of the IDH1 protein is present in the diseased cells. When IDH1 is present in this abnormal form, it produces too much 2-hydroxyglutarate (2-HG), a substance present in low levels in normal cells. When there is too much 2-HG in normal cells, immature cells may be unable to function normally, which may result in chondrosarcoma. AG-120 may stop the formation of abnormal IDH1 protein, which may reduce 2-HG levels in diseased cells to levels like those in normal cells. AG-120 targets only the abnormal IDH1 protein (and not the normal form).
    Location: 3 locations

  • Ivosidenib and Nivolumab for the Treatment of IDH1 Mutated Relapsed or Refractory Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome

    This phase II trial studies how well ivosidenib and nivolumab work for the treatment of IDH1 mutated acute myeloid leukemia that has come back (relapsed) or does not respond to treatment (refractory) or high risk myelodysplastic syndrome. IDH1 is a type of protein involved in metabolism (the process of providing your body’s cells with energy). In certain types of diseases such as acute myeloid leukemia, an abnormal form of the IDH1 protein is present in the diseased cells which produces an excess amount of a protein associated with cancer called 2-hydroxyglutarate (2-HG), which may keep cells from maturing into normal functioning cells and may cause them to become diseased. Ivosidenib is designed to block the abnormal IDH1 protein rather than the normal form of this protein and may reduce 2-HG levels. Cancer cells are able to “turn off” the immune system by increasing the production of a protein called PD-1. Nivolumab blocks PD-1 and may re-activate the immune response that may limit the progression of AML and MDS. Giving ivosidenib and nivolumab may work better in treating patients with acute myeloid leukemia or myelodysplastic compared to ivosidenib or nivolumab alone.
    Location: Yale University, New Haven, Connecticut

  • Ivosidenib and Venetoclax with or without Azacitidine in Treating Patients with IDH1 Mutated Hematologic Malignancies

    This phase Ib / II trial studies the side effects and best dose of venetoclax and how well it works when given together with ivosidenib with or without azacitidine, in treating patients with IDH1-mutated hematologic malignancies. Venetoclax and ivosidenib 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 ivosidenib and venetoclax with azacitidine may work better in treating patients with hematologic malignancies compared to ivosidenib and venetoclax alone.
    Location: 4 locations

  • Ivosidenib and Combination Chemotherapy for the Treatment of IDH1 Mutant Relapsed or Refractory Acute Myeloid Leukemia

    This phase I trial studies the side effects and best dose of ivosidenib when given together with combination chemotherapy for the treatment of 1DH1 mutant acute myeloid leukemia that has come back (relapsed) or does not respond to treatment (refractory). Ivosidenib may stop the growth of cancer cells by blocking the IDH1 mutation and some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as fludarabine phosphate, cytarabine, and filgrastim, 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 ivosidenib with combination chemotherapy may work better in treating patients with acute myeloid leukemia compared to chemotherapy alone.
    Location: Northwestern University, Chicago, Illinois

  • CPX-351 and Ivosidenib for the Treatment of IDH1 Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

    This phase II trial investigates how well CPX-351 and ivosidenib work in treating patients with acute myeloid leukemia or high-risk myelodysplastic syndrome that has IDH1 mutation. The safety of this drug combination will also be studied. IDH1 is a type of genetic mutation (change). Chemotherapy drugs, such as CPX-351, 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. Ivosidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. The purpose of this trial is to learn if CPX-351 in combination with ivosidenib can help to control IDH1-mutated acute myeloid leukemia or high-risk myelodysplastic syndrome.
    Location: M D Anderson Cancer Center, Houston, Texas

  • Gemcitabine and Cisplatin with Ivosidenib or Pemigatinib for the Treatment of Unresectable or Metastatic Cholangiocarcinoma

    This trial studies the side effects and best dose of gemcitabine and cisplatin when given together with ivosidenib or pemigatinib in treating patients with cholangiocarcinoma that cannot be removed with surgery (unresectable) or has spread to other places in the body (metastatic). Drugs used in chemotherapy, such as gemcitabine and cisplatin, 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. Ivosidenib and pemigatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving gemcitabine and cisplatin with ivosidenib or pemigatinib may work better in treating patients with cholangiocarcinoma compared to gemcitabine and cisplatin alone.
    Location: 3 locations