Evidence map›Paper›PMID 38445964›Full record

Trial reportNeuro-oncology2024

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

Isabel Arrillaga-Romany, Andrew Lassman, Susan L McGovern, Sabine Mueller, Burt Nabors, Martin van den Bent, Michael A Vogelbaum, Joshua E Allen, Allen S Melemed, Rohinton S Tarapore and 2 more

Registry-linked trialOpen access · hybridAbstract readRandomized Controlled TrialClinical Trial, Phase IIIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Neuro-oncology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05580562 (ONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy), which is not on this map. Cited by 43 papers.

0numbers the graph read from it
0cells of the map it votes in
43citing papers in PubMed
20.1field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT05580562 phase3recruitingnot on this map

ONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study

TypeinterventionalSponsorJazz PharmaceuticalsRan2023 to 2028Enrolled510ConditionsH3 K27M, GliomaArmsDordaviprone (ONC201), Dordaviprone (ONC201) + Placebo, Placebo
3 · Its place in the literature

Who cites it

43 citing papers in PubMed, 47 citations in OpenAlex.

  1. Trial
  2. Review
  3. Bridging pediatric and adult neuro-oncology: Insights into adolescents and young adults (AYA) central nervous system tumors.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026
    Review
  4. Article
  5. Article
  6. Review
  7. Review
  8. Review
  9. Review
  10. Article
  11. Recent Advances in Targeted Therapies for Adult Gliomas.Current neurology and neuroscience reports · 2026
    Review
  12. Targeted therapies in pediatric oncology: A start.Molecular therapy. Oncology · 2026
    Review
  13. Review
  14. Review
  15. Article
  16. Article
  17. Article
  18. Review
  19. Article
  20. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

12 authors at 10 institutions in 2 countries.

Isabel Arrillaga-RomanyMass General Cancer Center, Neuro-Oncology, Massachusetts General Hospital, Boston, Massachusetts, USA.ORCID 0000-0001-5951-1225
Andrew LassmanColumbia University Vagelos College of Physicians and Surgeons, Herbert Irving Comprehensive Cancer Center, New York-Presbyterian Hospital, New York City, New York, USA.
Susan L McGovernDepartment of Radiation Oncology, University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Sabine MuellerHelen Diller Family Comprehensive Cancer Center, University of California, San Francisco, San Francisco, California, USA.
Burt NaborsDepartment of Neuro-Oncology, University of Alabama at Birmingham, Birmingham, Alabama, USA.
Martin van den BentBrain Tumor Center at Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Michael A VogelbaumMoffitt Cancer Center, Tampa, Florida, USA.
Joshua E AllenChimerix, Inc., Durham, North Carolina, USA.
Allen S MelemedChimerix, Inc., Durham, North Carolina, USA.
Rohinton S TaraporeChimerix, Inc., Durham, North Carolina, USA.
Patrick Y WenCenter For Neuro-Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.
Timothy CloughesyBowyer Oncology Center, University of California Los Angeles, Los Angeles, California, USA.
Chimerix (United States) · USCenter for Neuro-Oncology · USErasmus MC Cancer Institute · NLMassachusetts General Hospital · USMoffitt Cancer Center · USNewYork–Presbyterian Hospital · USThe University of Texas MD Anderson Cancer Center · USUniversity of Alabama at Birmingham · USUniversity of California, Los Angeles · USUniversity of California, San Francisco · US

Funding

UCLA SPORE in Brain CancerP50CA211015 · NCI · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Yvonne Yu-Hsuan Chen · 2017 to 2026
$25.2M
ChimerixNCI NIH HHS P50 CA211015
6 · The paper itself

Abstract

backgroundH3 K27M-mutant diffuse glioma primarily affects children and young adults, is associated with a poor prognosis, and no effective systemic therapy is currently available. ONC201 (dordaviprone) has previously demonstrated efficacy in patients with recurrent disease. This phase 3 trial evaluates ONC201 in patients with newly diagnosed H3 K27M-mutant glioma.

methodsACTION (NCT05580562) is a randomized, double-blind, placebo-controlled, parallel-group, international phase 3 study of ONC201 in newly diagnosed H3 K27M-mutant diffuse glioma. Patients who have completed standard frontline radiotherapy are randomized 1:1:1 to receive placebo, once-weekly dordaviprone, or twice-weekly dordaviprone on 2 consecutive days. Primary efficacy endpoints are overall survival (OS) and progression-free survival (PFS); PFS is assessed by response assessment in neuro-oncology high-grade glioma criteria (RANO-HGG) by blind independent central review. Secondary objectives include safety, additional efficacy endpoints, clinical benefit, and quality of life. Eligible patients have histologically confirmed H3 K27M-mutant diffuse glioma, a Karnofsky/Lansky performance status ≥70, and completed first-line radiotherapy. Eligibility is not restricted by age; however, patients must be ≥10 kg at time of randomization. Patients with a primary spinal tumor, diffuse intrinsic pontine glioma, leptomeningeal disease, or cerebrospinal fluid dissemination are not eligible. ACTION is currently enrolling in multiple international sites.

Indexed as

Brain NeoplasmsGliomaMutationAdolescentAdultAgedChildDouble-Blind MethodFemaleFollow-Up StudiesHistonesHumansMaleMiddle AgedPrognosisQuality of LifeHistonesdiffuse gliomadordaviproneH3 K27MONC201Phase 3

Identifiers

PMID38445964
PMCPMC11066938
OpenAlexW4392490221

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.