ONC201 Clinical Trials - 13 Trials Found


Glioblastoma (GBM) and H3 K27M-mutant Glioma
NCT02525692 - Phase 2 - Interventional

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.

Recruiting

Male & Female

18 +


H3 K27M-mutant High-Grade Glioma
NCT03295396 - Phase 2 - Interventional

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.

Recruiting

Male & Female

18 +


H3 K27M-mutant High-Grade Glioma incl. DIPG
NCT03416530 - Phase 1 - Interventional

This is a multicenter, open-label, two 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.

Recruiting

Male & Female

2 - 18

years old


Neuroendocrine Tumors
NCT03034200 - Phase 2 - Interventional

The purpose of this study is to learn if a new drug, ONC201 can make tumors become smaller or go away completely. Investigators also want to learn if ONC201 can prevent new deposits of cancer from appearing in new places in participants (metastases). A phase 2 study of ONC201 in PC-PG (pheochromocytoma-paraganglioma) and other neuroendocrine tumors will determine whether inhibition of DRD2 (a member of the dopamine receptor family) is safe in neuroendocrine cancers including PC-PG. ONC201 is an investigational (experimental) agent and has a favorable safety profile in phase 1 and early phase 2 clinical trials in advanced cancers. This study design has been chosen to see whether ONC201 is associated with reduction of anti-hypertension medications, safety and significant efficacy against neuroendocrine tumors, especially PC-PG.

Recruiting

Male & Female

18 +


Type II Endometrial Cancer
NCT03485729 - Phase 2 - Interventional

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.

Recruiting

Female

18 +


Endometrial Cancer
NCT03099499 - Phase 2 - Interventional

ONC201 is a small molecule which selectively targets the G protein-coupled receptor DRD2. Downstream of target engagement, ONC201 activates the integrated stress response (ISR) in tumor cell leading to inactivation of Akt and extracellular signal-regulated kinase (ERK) signaling as well as induction of the TRAIL pathway. ONC201 also inhibits dopamine receptor 2 (DRD2), resulting in anti-tumor responses in preclinical models. Single agent ONC201 has been examined in open-label Phase I studies in patients with advanced, treatment refractory solid malignancies. Due to its differential anti-proliferative and pro-apoptotic response in tumor cells, treatment was overall well tolerated, and the recommended phase II dose of ONC201 was set at 625mg every three weeks. An additional dose-escalation phase I study (NCT02609230) is further evaluating weekly versus three week dosing in patients with advanced solid tumors and multiple myeloma. Preliminary data from these phase I studies suggests a possible clinical benefit in patients with advanced, chemo-refractory endometrial cancers, with at least one mixed response noted in a patient with clear cell histology. Hypothesis: Single agent ONC201 will demonstrate clinical benefit in women with recurrent or metastatic endometrial cancers, especially in those women with alterations in the Phosphoinositide 3 kinase (PI3K)/Akt/mammalian target of Rapamycin (mTOR) pathway.

Recruiting

Female

18 +


Breast Cancer and Endometrial Carcinoma
NCT03394027 - Phase 2 - Interventional

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: <TAB>Medical history <TAB>Physical exam <TAB>Heart, blood, and urine tests <TAB>CT and bone scans <TAB>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.

Recruiting

Male & Female

18 - 99

years old


Multiple Myeloma
NCT03492138 - Phase 1 phase 2 - Interventional

ONC201 is a novel dopamine receptor D2 antagonist that is able to activate the integrated stress response pathway. It is active against multiple myeloma cells in vitro, both as a single agent and in combination with corticosteroids and proteasome inhibitors. In order to document superiority over the combination compared to the individual agents of ixazomib and ONC201 in a single arm study, there will initially be a run-in period of weekly ONC201 625 mg with dexamethasone 40 mg such that if there is progression of disease (25% increase) after 4 weeks or less than a minimal response (25% reduction) after 8 weeks then ixazomib will be added. Dexamethasone is dose-reduced to 20 mg at the same schedule for subjects ≥ 75 years old. If patients do achieve single-agent responses with ONC201 (minimal response or better), they will continue with weekly ONC201 and dexamethasone until progression, with response assessments after each 28-day cycle. Patients who have previously been treated on another clinical trial with weekly ONC201 625mg with dexamethasone with progression while receiving treatment do not need to complete the run-in phase of the study. At the time of progression, they will proceed to the 3 drug combination phase of the study. It is at the point of 3 drug initiation, that below phase I DLT principles or phase II disease control rate considerations apply.

Recruiting

Male & Female

18 +


Multiple Myeloma
NCT02863991 - Phase 1 phase 2 - Interventional

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

Recruiting

Male & Female

18 +


Solid Tumors
NCT02609230 - Phase 1 - Interventional

This study will determine the maximum tolerated dose (MTD) and/or the recommended phase II dose (RP2D) of single agent ONC201 in patients with advanced solid tumors or multiple myeloma.

Recruiting

Male & Female

18 +


Solid Tumors
NCT02250781 - Phase 1 - Interventional

This phase I trial studies the side effects and best dose of Oral ONC201 in treating patients with advanced solid tumors. Oral ONC201 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Suspended

Male & Female

18 +


Lymphoma
NCT02420795 - Phase 1 phase 2 - Interventional

This clinical research study will be done in two parts. The goal of the first part is to find the highest tolerable dose of ONC201 that can be given to patients with relapsed or refractory MCL, DLBCL, and TLCL. Groups of subjects will receive increasing doses of ONC201 by mouth on day 1 of each 21 day cycle or on Day 1 of every week until there are side effects that are not tolerated or a maximum of 625 mg has been found to be tolerable. As a result of new information, it has been decided that subjects in Arm A will continue receiving their dosing every 3 weeks. All other subjects will be dosed weekly. The dose you receive may be too low to have an effect or so high it causes bad side effects. In the second part of this study the highest dose of NC201 will be given to learn if ONC201 can help to control the disease. Please note that this is the first time ONC201 will be given to human subjects.

Recruiting

Male & Female

18 +


Leukemia
NCT02392572 - Phase 1 phase 2 - Interventional

The goal of Phase I of this clinical research study is to find the highest tolerable dose of ONC201 alone or in combination with low dose cytarabine (LDAC) that can be given to patients with relapsed or refractory AML, ALL, or MDS. The goal of Phase II of this study is to learn if the dose of ONC201 given alone or in combination with LDAC that is found in Phase I can help to control the disease. The safety of the study drug will be studied in both phases of this study. This is the first study using ONC201 in humans. ONC201 given alone or in combination with LDAC is in a very early stage of development for use in humans. Providing direct medical benefit to you is not the purpose of this study. While Phase II will look at the effectiveness of the study drug given alone or in combination with LDAC, the main purpose of this study is to learn about the safety of the drug.

Recruiting

Male & Female

18 +


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