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NCT04629209

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

Withdrawn Phase 2 Last updated 24 November 2023
What this trial tests

Phase 2 trial testing ONC201 in Glioblastoma. Withdrawn.

Timeline
28 June 2024
Primary endpoint
31 December 2024
30 June 2025

Quick facts

Lead sponsorMasonic Cancer Center, University of Minnesota
PhasePhase 2
StatusWithdrawn
Study typeINTERVENTIONAL
Allocationnon randomized
Designparallel
Maskingnone
Primary purposetreatment
Start date28 June 2024
Primary completion31 December 2024
Estimated completion30 June 2025
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

Masonic Cancer Center, University of Minnesota

Who can join

18 and older, any sex, with Glioblastoma. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is an open-label, two arm study. The trial will enroll a total of up to 36 patients. Arm A will enroll up to a total of 6 evaluable patients and Arm B will enroll up to a total of 30 evaluable patients. Arm A will explore the intra-tumoral ONC201 concentrations and pharmacodynamic activity in adult EGFR-low glioblastoma patients. Arm B will determine the radiographic efficacy of ONC201 in adult recurrent EGFR-low glioblastoma patients. All patients will be treated with oral ONC201 (625 mg) twice weekly, 2 consecutive days on and 5 days off per week schedule.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Mechanisms of imipridones in targeting mitochondrial metabolism in cancer cells.
    Bonner ER, Waszak SM, Grotzer MA, Mueller S, et al · · 2021 · cited 50× · PMID 33336683 · DOI 10.1093/neuonc/noaa283
  2. Metabolic Rewiring in Glioblastoma Cancer: <i>EGFR</i>, <i>IDH</i> and Beyond.
    El Khayari A, Bouchmaa N, Taib B, Wei Z, et al · · 2022 · cited 43× · PMID 35912242 · DOI 10.3389/fonc.2022.901951
  3. A review of current therapeutics targeting the mitochondrial protease ClpP in diffuse midline glioma, H3 K27-altered.
    Jackson ER, Persson ML, Fish CJ, Findlay IJ, et al · · 2024 · cited 30× · PMID 37589388 · DOI 10.1093/neuonc/noad144
  4. Hijacking of the nervous system in cancer: mechanism and therapeutic targets.
    Zhang Y, Liao Q, Wen X, Fan J, et al · · 2025 · cited 20× · PMID 39915765 · DOI 10.1186/s12943-025-02246-5
  5. Imipridones and Dopamine Receptor Antagonism in the Therapeutic Management of Gliomas.
    Burton E, Ozer BH, Boris L, Brown D, et al · · 2024 · cited 3× · PMID 38868646 · DOI 10.1016/j.yao.2024.02.003
  6. Targeting the Mitochondria in High-Grade Gliomas.
    Sathe S, Li Q, Jung J, Wu J. · · 2025 · cited 2× · PMID 41008904 · DOI 10.3390/cancers17183062
  7. The GPCR Connection: Linking Alzheimer's Disease and Glioblastoma.
    Caniceiro AB, Agostinho SP, Piochi LF, Rosário-Ferreira N, et al · · 2026 · PMID 41958080 · DOI 10.1111/jcmm.71131

Verify or expand the search:

Other trials of ONC201

Trials testing the same drug.

Other recruiting trials for Glioblastoma

Currently open trials in the same condition.

Other Masonic Cancer Center, University of Minnesota trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

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