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S-Gboxin (lyophilized form of S-Gboxin) for the treatment diffuse midline glioma with H3 K27M / H3 K28M mutation

S-Gboxin

Andrey Petrov, Dr

Oliever Parteny

F. 50y.o. 77kg, DS confirmed 18 Nov. 2022 Received treatment: 11 courses of TMZ (240mg) Radiotherapy 30 courses of 1.8Gr (total 54 Gr, TMZ 140 mg on RT). Bevacizumab intravenously -400 mg -0-14-28-42 (December-February 2023). Severe side effects. The attending physician discontinued bevacizumab after 4 courses. In remission for 11 months. According to the results of the MRI dated April 28, 2024, continued growth.

Neurological status is good. Reflexes are not impaired. To date, treatment options have been exhausted. Participation of the patient in Clinical Trials is impossible. As of April 2024, negative dynamics. The patient signed an informed consent agreement. Information on taking L-S-Gboxin (Lyophilisate) as part of a compassionate program - explained.

Written consent of the attending physician for the patient's participation in the Compassion Program (TRIANGLE) with L-S-Gboxi (Lyophilisate) - received.

(medical documentation will be available on clinicaltrials.gov within a month from the date of publication - patient's consent to publish the medical history and examination results has been obtained)

TREATMENT PLAN

Оn days 1 to 3 of treatment, the patient will receive a sublingual form of the lyophilized form of L-S-Gboxin at a dose of 5 mg/kg.

If there is no toxicity, on days 4 to 14 the patient will receive a sublingual form of the lyophilized form of S-Gboxin at a dose of 10 mg/kg.

The first course of treatment is 14 days. On day 20, MRI + PET-CT monitoring is carried out to assess the dynamics of tumor growth. Based on the results of the examination plan, the attending physician and the main investigator make a decision on the continuation of treatment and the duration of the second course of therapy.

A Single Patient Compassionate Use Clinical Trial for the Use of L-S-Gboxin (Lyophilized Form of S-Gboxin) for the Treatment Diffuse Midline Glioma, H3 K27/28-altered (DMG), Including Diffuse Intrinsic Pontine Glioma (DIPG)

Petrov, Andrey

Antineoplastic Agents

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

ONC201 (dordaviprone) is a ClpP agonist and DRD2 antagonist.

ONC201 (dordaviprone)

Rohinton Tarapore, PhD

Madelyn Cooper

Ramya Tadipatri, MD

Hema V Buddha

Richard M Green, MD

Mariko Sato

Megan Paul, MD

Ashley Mettetal

Kathleen Dorris, MD

Lindsay Kilburn, MD, MS

Yazmin Odia, MD, MS

Anais Vega

Nikki Pope

Petros Nikolinakos, MD

Andrea Siu, MPH

Kelley Hutchins, DO

Angela Waanders, MD, MPH

Misty Gravelin

Carl Koschmann, MD, MS

Clark Chen, MD, PhD

Alison Barnwell

Nicole Shonka

Robert Aiken, MD

Anna Yaffe

Sharon Gardner, MD

Jennifer Serventi, PA

Sara Guedry

Alison Patterson

Teresa Hannah

Nazanin Majd

Wafik Zaky

Joe Mendez

This is an intermediate-size expanded access protocol to provide ONC201 (dordaviprone) to patients with H3 K27M-mutant and/or midline gliomas who cannot access ONC201 (dordaviprone) through clinical trials.

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

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Single Patient Compassionate Use Clinical Trial for the Use of L-S-Gboxin (Lyophilized Form of S-Gboxin) for the Treatment Diffuse Midline Glioma, H3 K27/28-altered (DMG), Including Diffuse Intrinsic Pontine Glioma (DIPG)	NCT06439849	DIPG Brain Tumo... Diffuse Brainst... H3 K27M H3 K28M GBM	S-Gboxin	-	Petrov, Andrey
ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)	NCT05580562	H3 K27M Glioma	ONC201 ONC201 + Placeb... Placebo	-	Chimerix
Intermediate-size Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline Gliomas	NCT04617002	Glioma H3 K27M	ONC201 (dordavi...	0 Years -	Chimerix
Treatment of Relapsed/Refractory Intracranial Glioma in Patients Under 22 Years of Age	NCT05956821	Glioblastoma Mu... Anaplastic Astr... Fibrillary Astr... Oligodendroglio... Diffuse Intrins... Diffuse Intrins... DIPG Brain Tumo... H3 K27M	SIACI of cetuxi...	1 Year - 21 Years	University of Miami
Treatment of Relapsed/Refractory Intracranial Glioma in Patients Under 22 Years of Age	NCT05956821	Glioblastoma Mu... Anaplastic Astr... Fibrillary Astr... Oligodendroglio... Diffuse Intrins... Diffuse Intrins... DIPG Brain Tumo... H3 K27M	SIACI of cetuxi...	1 Year - 21 Years	University of Miami

Spots Global Cancer Trial Database for h3 k27m

Every month we try and update this database with for h3 k27m cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for h3 k27m

Every month we try and update this database with for h3 k27m cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for h3 k27m cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.