Clinical Trials Using Akt/ERK Inhibitor ONC201

Clinical trials are research studies that involve people. The clinical trials on this list are studying Akt/ERK Inhibitor ONC201. 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 1-13 of 13
  • ONC201 in Pediatric H3 K27M Gliomas

    This is a multicenter, open-label, five arm, dose escalation, phase I study of oral ONC201 in pediatric patients with newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) and recurrent / refractory H3 K27M gliomas. Arm A will define the RP2D for single agent ONC201 in pediatric patients with glioma who are positive for the H3 K27M mutation (positive testing in CLIA laboratory) and have completed at least one line of prior therapy. This will allow for recurrent patients and also patients who have not yet recurred, but have completed radiation and will inevitably recur based on prior clinical experience and the literature. Arm B will define the RP2D for ONC201 in combination with radiation in pediatric patients with newly diagnosed DIPG. Arm C will determine intratumoral drug concentrations and biomarker expression in pediatric patients with midline gliomas. Arm D will determine H3 K27M DNA levels and drug concentrations in the CSF of pediatric H3 K27M-mutant glioma patients. Arm E will determine the RP2D for single agent ONC201 administered as a liquid formulation in Ora-Sweet to patients with DIPG and / or H3 K27M glioma. All patients must be 2-12 weeks from completion of first-line radiation.
    Location: 5 locations

  • ONC201 in Adults With Recurrent H3 K27M-mutant Glioma

    The primary objective of this phase II trial is to determine the efficacy and safety of ONC201, an oral small molecule imipridone DRD2 antagonist, in adult subjects with recurrent high-grade glioma. This study will test the research hypothesis that histone H3 K27M mutation sensitizes to oral administration of ONC201 in gliomas.
    Location: 5 locations

  • ONC201 in Recurrent or Metastatic Type II Endometrial Cancer Endometrial Cancer

    This is a a Simon's two-stage, non-randomized, open label, 2-arm Phase II trial of ONC201 in women with metastatic or recurrent Type II endometrial cancer who failed at least 1 prior chemotherapy regimen.
    Location: 4 locations

  • Akt / ERK inhibitor ONC201 in Treating Patients with Recurrent or Metastatic Endometrial Cancer

    This phase II trial studies how well Akt / ERK inhibitor ONC201 works in treating patients with endometrial cancer that has come back or has spread to other places in the body. Akt / ERK inhibitor ONC201 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
    Location: 2 locations

  • Oral ONC201 in Relapsed / Refractory Multiple Myeloma

    ONC201 is an orally bioavailable first-in-class small molecule with demonstrated antitumor activity in preclinical models of difficult-to-treat solid and liquid tumors without imparting significant toxicity. This is a Phase 1 / 2 open-label study of ONC201 administered orally once every week in combination with dexamethasone to patients with relapsed / refractory multiple myeloma. Funding Source - FDA OOPD
    Location: 2 locations

  • Oral ONC201 in Recurrent GBM, H3 K27M Glioma, and Midline Glioma

    ONC201 is a new drug candidate that kills cancer cells but not normal cells in laboratory studies and has been previously evaluated in a phase I clinical trial in advanced cancer patients. This clinical trial will enroll patients with recurrent glioblastoma or recurrent WHO Grade IV gliomas with the H3 K27M mutation.
    Location: 4 locations

  • ONC201 with or without Methionine-Restricted Diet in Treating Patients with Metastatic or Unresectable Triple Negative Breast Cancer

    This phase II trial studies how well Akt / ERK inhibitor ONC201 (ONC201) with or without methionine-restricted (MR) diet works in treating patients with triple negative breast cancer that has spread to other places in the body or cannot be removed by surgery. ONC201 activates a process that leads to the death of a cell. ONC201 is able to target tumor cells to get rid of them, but does not affect normal cells. Patients on the MR diet eat only methionine-restricted food. The addition of an intermittent MR diet may enhance the activity of ONC201. Giving ONC201 and an MR diet may work better in treating patients with breast cancer.
    Location: University of Wisconsin Hospital and Clinics, Madison, Wisconsin

  • Ixazomib Citrate, ONC201, and Dexamethasone in Treating Participants with Recurrent and / or Refractory Multiple Myeloma

    This phase I / II trial studies the side effects and best dose of ixazomib citrate when given together with Akt / ERK inhibitor ONC201 (ONC201) and dexamethasone in treating participants with multiple myeloma that has come back and / or does not respond to treatment. Ixazomib citrate and ONC201 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as dexamethasone, 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 ixazomib citrate, ONC201, and dexamethasone may work better in treating participants with multiple myeloma.
    Location: Icahn School of Medicine at Mount Sinai, New York, New York

  • ONC201 in Recurrent / Refractory Metastatic Breast Cancer and Advanced Endometrial Carcinoma

    Background: The new drug ONC201 have been shown to kill breast cancer and endometrial cancer cells in the laboratory. The exact mechanism of action is not completely clear yet, but the ONC201 destroys the mitochondria inside the cells. Blocking mitochondrial activity may kill tumor cells, which would shrink tumors. Researchers want to see if ONC201 helps shrink tumors of certain breast or endometrial cancers and if that effect is maintained. Objective: To see if ONC201 shrinks tumors with a lasting effect. Eligibility: Adults ages 18 and older who have metastatic breast cancer (hormone-positive or triple-negative) or metastatic endometrial cancers. Design: Participants will be screened with: Medical history Physical exam Heart, blood, and urine tests CT and bone scans Review of medical report and tumor sample Participants will have a tumor biopsy before starting treatment and after 5 weeks taking the study drug. A scan or ultrasound may be used to guide the biopsy. Patients will receive local anesthetic and a needle will remove a small piece of tumor. The study will be done in 28-day cycles. Every day 1 of each cycle participants will repeat most screening tests, will be seen by the physician and receive a supply of the study drug. Participants will take the study drug by mouth once every 7 days. They will keep a diary of when they take the drug and any side effects. During cycle 1, participants will get weekly calls to discuss their health and symptoms. Images will be repeated every 2 cycles to evaluate reponse to the treatment. ...
    Location: National Institutes of Health Clinical Center, Bethesda, Maryland

  • Akt / ERK Inhibitor ONC201 in Treating Patients with Neuroendocrine Tumors That Are Locally Advanced, Metastatic, Recurrent, Refractory, or Cannot Be Removed by Surgery

    This phase II trial studies how well akt / ERK inhibitor ONC201 works in treating patients with neuroendocrine tumors that have spread to other places in the body, have come back, do not respond to treatment, or cannot be removed by surgery. Akt / ERK inhibitor ONC201 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
    Location: Case Comprehensive Cancer Center, Cleveland, Ohio

  • Akt / ERK Inhibitor ONC201 in Treating Patients with Advanced Solid Tumors or Multiple Myeloma

    This phase I trial studies the side effects and the best dose of v-akt murine thymoma viral oncogene homolog 1 (Akt) / extracellular signal-regulated protein kinase (ERK) inhibitor ONC201 in treating patients with advanced solid tumors or multiple myeloma. Akt / ERK Inhibitor ONC201 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
    Location: Fox Chase Cancer Center, Philadelphia, Pennsylvania

  • Akt / ERK Inhibitor ONC201 in Treating Patients with Relapsed or Refractory Non-Hodgkin's Lymphoma

    This phase I / II trial studies the side effects and the best dose of v-akt murine thymoma viral oncogene homolog (Akt) / mitogen-activated protein kinase 1(ERK) inhibitor ONC201 and to see how well it works in treating patients with non-Hodgkin's lymphoma that has returned after a period of improvement or does not respond to treatment. Akt / ERK inhibitor ONC201 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: M D Anderson Cancer Center, Houston, Texas

  • ONC201 in Treating Patients with Relapsed or Refractory Acute Leukemia or High-Risk Myelodysplastic Syndrome

    This phase I / II trial studies the side effects and best dose of ONC201 and to see how well it works in treating patients with acute leukemia or high-risk myelodysplastic syndrome that has returned after a period of improvement (relapsed) or does not respond to treatment (refractory). ONC201 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    Location: M D Anderson Cancer Center, Houston, Texas