Neoplasms

All Conditions

Rare Diseases

Glioma

Neoplasms, Neuroepithelial

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Neuroepithelioma

Antineoplastic Agents

All Drugs and Chemicals

TIC10 compound

Participants ≥ 52.5 kg will receive 625 mg of ONC201 (5 × 125-mg capsules) dosing days; participants \< 52.5 kg will receive a dose (and corresponding number of capsules) scaled by body weight and rounded to 125-mg increments.

ONC201

Participants ≥ 52.5 kg will receive 625 mg of ONC201 (5 × 125-mg capsules) or matching placebo on dosing days; participants \< 52.5 kg will receive a dose (and corresponding number of capsules) scaled by body weight and rounded to 125-mg increments

ONC201 + Placebo

Participants will receive placebo (same number of capsules as the ONC201 dose) on dosing days

Placebo

Tarapore, PhD

Robert Yoo, DO

Na Tosha Gatson

Lindsey Hoffmann, DO

Maciej Mrugala, MD

Xiao-Tang Kong, MD, PhD

Jessica Schulte, MD

Richard Green, MD

Phioanh Nghiemphu, MD

Mariko Sato, MD, PhD

Nicholas Butowski, MD

Akanksha Sharma, MD

Kate Therkelsen, MD

Nicholas Blondin, MD

Edina Komlodi-Pasztor, MD, PhD

Kurt Jaeckle, MD

Yazmin Odia, MD

Paul R Ferguson

Trisha Larkin, MD

Michael Vogelbaum, MD

Tobey MacDonald, MD

Kelley Hutchins, DO, MPH

Daniel Runco, MD

David Dickens, MD

Kaylyn Sinicrope, MD

Caroline Goldin, MD

Lily Pham, MD

Isabel Arrillaga-Romany, MD

Patrick Wen, MD

Carl Koschmann, MD

Elizabeth Neil, MD

Michael Ruff, MD

Andrew Cluster, MD

Chittalsinh Raulji, MD

Shama Farooq, MD

Robert Aiken

Lauren Weintraub, MD

Adilia Hormigo, MD, PhD

Aya Haggiagi

Luca Szalontay, MD

David Korones, MD

Ashley Sumrall, MD

Margaret Johnson, MD, MPH

Peter de Blank, MD

Christine Cordova, MD

Shirley Ong, MD

Maya Hrachova, DO

Prakash Ambady

Prakash Ambady, MB, MD

Stephen Bagley, MD

Nina Martinez, MD

Frank Lieberman, MD

Sage Green, DO, MD

Karen Fink, MD, PhD

Daniel Bowers, MD

Evguenia Gachimova (Adults

Wafik Zaky (Pediatrics)

Nazanin Majd (Adults)

Jay-Jiguang Zhu, MD

Andrew Brenner, MD

Rachel Kingsford

Yuri Kida

Joe Mendez, MD

Adam Cohen, MD

Melissa Mark, MD

Tresa McGranahan, MD

Ankush Bhatia, MD

David Ziegler, BS, MB, MD

Helen Wheeler, MD

Zarnie Lwin, MD

John Heath

Lawrence Cher

Santosh Valvi

Anna Bergmeister-Berghoff, MD

Johannes Gojo

Paula De Robles, MD

Rebecca Harrison, MD

George Michaiel

Seth Climans, MD

Donna Johnston, MD

Mary Lim-Fat, MD

Julie Bennett, MD

Sarah Lapointe

Søren Møller, MD

Charlotte Aaquist Haslund, MD

Rikke Dahlrot, MD

Cornelis van Tilburg, MD, PhD

Antje Wick, MD

Michael Frühwald, MD

Peter Hau, MD

Michael Ronellenfitsch, MD

Ulrich Herrlinger, MD

Martin Glas, MD

Roland Goldbrunner, MD, PhD

Andreas Jödicke, MD

Katharina Sahm, MD

Sylvia Kurz, MD

Michal Yalon, MD, PhD

Abed El Salem Abu Quider, MD

Liat Oren

Alexander Lossos, MD

Helen Toledano, MD

Shlomit Yust-Katz, MD

Deborah Blumenthal, MD

This is a randomized, double-blind, placebo-controlled, parallel-group, international, Phase 3 study in patients with newly diagnosed H3 K27M-mutant diffuse glioma to assess whether treatment with ONC201 following frontline radiotherapy will extend overall survival and progression-free survival in this population. Eligible participants will have histologically diagnosed H3 K27M-mutant diffuse glioma and have completed standard frontline radiotherapy.

ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)

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

Overall Survival is defined as the time from randomization to death due to any cause.

PFS is defined as time from randomization to disease progression (PD) or death.

Incidence of overall, treatment-related, Grade 3 or higher in severity, serious, fatal, those resulting in treatment discontinuation, and events of special interest

Percentage of participants with clinically significant laboratory results

PFS using RANO-HGG criteria for participants with measurable contrast-enhancing disease

Corticosteroid response will be measured by a confirmed 50% decrease in use of dexamethasone or equivalent

Performance status response will be measured by confirmed increase in Karnofsky Performance Status (KPS) or Lansky Performance Status (LPS)

Chimerix

Spots Global Cancer Trial Database for onc201 + placebo