ONC201

DRUG14 trials

Sponsors

Prinses Maxima Centrum voor Kinderoncologie B.V., Jazz Pharmaceuticals, Peter Anderson, Fox Chase Cancer Center, National Cancer Institute (NCI)

Conditions

Advanced Colorectal CancerAdvanced GlioblastomaAdvanced Non-small Cell Lung CancerAdvanced Triple-negative Breast CancerCarcinosarcomaClear Cell Endometrial CarcinomaDiffuse Intrinsic Pontine GliomaDiffuse Midline Glioma, H3 K27M-Mutant

Phase 1

A First-in-man Phase I/II Study of Oral ONC201 in Patients With Advanced Cancer

WithdrawnNCT02038699

Jazz PharmaceuticalsAdvanced Colorectal Cancer, Advanced Glioblastoma, Advanced Non-small Cell Lung Cancer +1

Start: 2014-01-31End: 2016-12-31Updated: 2021-04-30

Oral ONC201 in Relapsed/Refractory Multiple Myeloma

TerminatedNCT02863991

Jazz PharmaceuticalsMultiple Myeloma

Start: 2017-04-19End: 2019-12-16Updated: 2024-07-03

Ixazomib, ONC201, and Dexamethasone in Relapsed/Refractory Multiple Myeloma

TerminatedNCT03492138

Ajai ChariMultiple Myeloma

Start: 2018-03-26End: 2020-01-14Updated: 2020-05-29

ONC201 and Atezolizumab in Obesity-Driven Endometrial Cancer

RecruitingNCT05542407

UNC Lineberger Comprehensive Cancer CenterCarcinosarcoma, Clear Cell Endometrial Carcinoma, Endometrial Cancer +5

Start: 2023-10-23End: 2028-07-31Target: 58Updated: 2026-01-29

Phase 2

Phase 2 Study of ONC201 in Neuroendocrine Tumors

CompletedNCT03034200

Peter AndersonMetastatic Neuroendocrine Tumor, Recurrent Neuroendocrine Tumor

Start: 2017-08-02End: 2023-05-19Updated: 2024-11-26

Single Agent ONC201 in Recurrent or Metastatic Endometrial Cancer

TerminatedNCT03099499

Fox Chase Cancer CenterEndometrial Cancer

Start: 2017-06-08End: 2020-09-09Updated: 2024-12-24

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

CompletedNCT03394027

National Cancer Institute (NCI)Endometrial Cancer, Hormone Receptor Positive, HER2 Negative Breast Cancer, Triple Negative Breast Cancer

Start: 2018-01-17End: 2021-10-07Updated: 2022-08-23

A Phase II, Open Label Study of ONC201 in Adults With EGFR-low Glioblastoma

WithdrawnNCT04629209

Masonic Cancer Center, University of MinnesotaGlioblastoma

Start: 2024-06-28End: 2025-06-30Updated: 2023-11-24

Combination Therapy for the Treatment of Diffuse Midline Gliomas

RecruitingNCT05009992

University of California, San FranciscoDiffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Recurrent Diffuse Intrinsic Pontine Glioma +3

Start: 2021-10-20End: 2029-06-30Target: 360Updated: 2025-12-17

A Study of ONC201 for Refractory Meningioma

WithdrawnNCT06012929

University of NebraskaMeningioma, Refractory Meningioma, Relapsed Meningioma

Start: 2026-12-01End: 2027-04-01Updated: 2026-01-26

PNOC022: A Combination Therapy Trial using an Adaptive Platform Design for Children and Young Adults with Diffuse Midline Gliomas (DMGs) including Diffuse Intrinsic Pontine Gliomas (DIPGs) at Initial Diagnosis, Post-Radiation Therapy and at Time of Progression

SuspendedCTIS2023-507598-16-00

Prinses Maxima Centrum voor Kinderoncologie B.V.Diffuse Midline Gliomas (DMGs) including Diffuse Intrinsic Pontine Gliomas (DIPGs)

Start: 2022-10-20Target: 30Updated: 2025-08-21

Phase 3

Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication 2.0

RecruitingNCT05476939

Gustave Roussy, Cancer Campus, Grand ParisDiffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3K27-altered, Diffuse Midline Glioma, H3 K27M-Mutant

Start: 2022-09-29End: 2031-09-01Target: 433Updated: 2026-02-05

Unknown Phase

Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline High Grade Gliomas

Jazz PharmaceuticalsGlioma

Updated: 2022-03-14

Expanded Access Use of ONC201 in a Patient With Diffuse Intrinsic Pontine Gliomas

Jazz PharmaceuticalsGlioma

Updated: 2022-05-26

Related Papers

EXTH-89. Precision Targeting of PI3K/mTOR in Diffuse Midline Glioma: GCT007 as a Novel Brain-Penetrant PIK3CA Inhibitor

Neuro-Oncology2025-11-01

Omnigene-Gut<sup>tm</sup> ensures fecal microbiome stability in the pediatric population.

2024-12-06

DIPG-12. ASSESSING THE EFFICACY OF BAY2402234 IN COMBINATION WITH ONC201 IN IMMUNE-COMPETENT MURINE MODELS OF DIFFUSE MIDLINE GLIOMA

Neuro-Oncology2024-06-18

Abstract 3173: Impact of hypoxia on the integrated stress response activated by imipridones ONC201 and ONC206 in pediatric diffuse midline glioma cells

Cancer Research2024-03-22

ACTION: a randomized phase 3 study of ONC201 (dordaviprone) in patients with newly diagnosed H3 K27M-mutant diffuse glioma

Neuro-Oncology2024-03-0640 citations

Rational combination platform trial design for children and young adults with diffuse midline glioma: A report from PNOC

Neuro-Oncology2023-12-202 citations

EXTH-36. COMBINING ONC201 AND PAXALISIB FOR THE TREATMENT OF DIFFUSE MIDLINE GLIOMA (DMG); THE PRECLINICAL RESULTS UNDERPINNING THE INTERNATIONAL PHASE II CLINICAL TRIAL (NCT05009992)

Neuro-Oncology2023-11-01

EXTH-31. EXPLOITING THE GENETIC DEPENDENCY ON PI3K/MTOR SIGNALING FOR THE TREATMENT OF H3-ALTERED DIFFUSE MIDLINE GLIOMA

Neuro-Oncology2023-11-01

A Single-Arm, Open-Label Phase II Study of ONC201 in Recurrent/Refractory Metastatic Breast Cancer and Advanced Endometrial Carcinoma

The Oncologist2023-06-059 citations

ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline Glioma

Cancer Research2023-05-0574 citations

CTNI-61. CLINICAL EFFICACY AND PREDICTIVE BIOMARKERS OF ONC201 IN H3K27M-MUTANT DIFFUSE MIDLINE GLIOMA

Neuro-Oncology2022-11-014 citations

EXTH-12. PRECLINICAL AND CASE STUDY EXAMINATION OF THE COMBINATION OF THE CLPP AGONIST ONC201 WITH THE PI3K/AKT INHIBITOR PAXALISIB FOR THE TREATMENT OF DIFFUSE MIDLINE GLIOMA

Neuro-Oncology2022-11-01

TMIC-06. ANTAGONISM OF DRD2 USING ONC201 INCREASED EXPRESSION OF ANTIGEN PRESENTATION PATHWAY PROTEINS IN DIFFUSE MIDLINE GLIOMA, RECRUITING TUMOR INFILTRATING LYMPHOCYTES IN VIVO

Neuro-Oncology2022-11-01

CTNI-24. SINGLE CENTER EXPERIENCE OF DOPAMINE ANTAGONIST ONC-201 FOR RECURRENT H3K27M-MUTANT GLIOBLASTOMA IN ADULTS

Neuro-Oncology2022-11-01

A Single-Center Experience of Dopamine Antagonist ONC201 for Recurrent Histone H3 Lysine 27-to-Methionine (H3K27M)-Mutant Glioblastoma in Adults.

2022-08-191 citations

CLRM-02 SINGLE CENTER EXPERIENCE OF DOPAMINE ANTAGONIST ONC-201 FOR RECURRENT H3K27M-MUTANT GLIOBLASTOMA IN ADULTS

Neuro-Oncology Advances2022-08-012 citations

DIPG-07. Preclinical and case study results underpinning the phase II clinical trial testing the combination of ONC201 and paxalisib for the treatment of patients with diffuse midline glioma (NCT05009992)

Neuro-Oncology2022-06-01

Targeting regulated cell death (RCD) with small-molecule compounds in triple-negative breast cancer: a revisited perspective from molecular mechanisms to targeted therapies.

2022-04-12102 citations

Imipridones affect tumor bioenergetics and promote cell lineage differentiation in diffuse midline gliomas

Neuro-Oncology2022-02-1191 citations

Phase II Study of ONC201 in Neuroendocrine Tumors including Pheochromocytoma-Paraganglioma and Desmoplastic Small Round Cell Tumor

Clinical Cancer Research2022-01-1130 citations

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