RecruitingPhase 1

ONC206 for Treatment of Newly Diagnosed, Recurrent Diffuse Midline Gliomas, and Other Recurrent Malignant CNS Tumors

United States, Netherlands, Switzerland208 participantsStarted 2021-08-23

Plain-language summary

This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors. ONC206 is a recently discovered compound that may stop cancer cells from growing. This drug has been shown in laboratory experiments to kill brain tumor cells by causing a so called "stress response" in tumor cells. This stress response causes cancer cells to die, but without affecting normal cells. ONC206 alone or in combination with radiation therapy may be effective in treating newly diagnosed or recurrent diffuse midline gliomas and other recurrent primary malignant CNS tumors.

Who can participate

Age range

2 Years – 21 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * ARM A: Children and young adults with DMG, H3K27 altered (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who completed at least one line of prior therapy. Prior treatment must have included focal radiation therapy and patients must be within 4-14 weeks from completion of radiation therapy to registration (patients must start treatment within 1 week from registration), have not started any other therapies post-radiation, and have no evidence of disease progression. * ARM A: Tumor tissue confirmation of DMG, H3K27 altered is mandatory and pathology must be consistent with a DMG, H3K27 altered. * ARM A: Participants must have recovered from all acute side effects of prior therapy. * ARM A: From the projected start of scheduled study treatment, the following time periods must have elapsed: 5 half-lives from any investigational agent, 4 weeks from cytotoxic therapy (except 23 days for temozolomide and 6 weeks from nitrosoureas), 4 weeks from antibodies and must be at least 7 days since the completion of therapy with a biologic or small molecule agent. For any biologic or small molecule agent with known adverse events that can occur beyond 7 days after administration, the period prior to enrollment must be beyond the time during which adverse events are known to occur (these should be discussed with the study team) * ARM B: Newly diagnosed children and young adults (Dose escalation: 2-21 years of age; Dose expansion: 2 years of …

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Since this is a Phase 1 trial primarily focused on finding the maximum tolerated dose of ONC206, what does that mean for what's currently known about whether it actually works against my specific tumor type?
2The trial includes both newly diagnosed diffuse midline gliomas and recurrent tumors — given my particular diagnosis and situation, would my doctor consider me a candidate worth discussing with the trial team, and how does my current treatment history affect that?
3Because the main thing being measured is dose-limiting toxicities, what kinds of side effects have been seen with ONC206 so far, and how would those be managed if I were participating?
4Is there a standard treatment option I should consider first before exploring a Phase 1 trial like this, or does my specific diagnosis make a trial like this worth discussing as an earlier option?
5What would the practical demands of joining this trial look like — how often would I need to come in for treatment or monitoring, and is that realistic given where I live and my current health?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Proportion of participants with dose-limiting toxicities (DLT)

Timeframe: Within 4 weeks after first dose

Maximum tolerated dose (MTD) of ONC206

Timeframe: Within 4 weeks after first dose

Trial details

NCT IDNCT04732065

SponsorSabine Mueller, MD, PhD

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-05-31

Contact for this trial

PNOC Operations

(415) 502-1600 PNOC023@ucsf.edu

View on ClinicalTrials.gov More Diffuse Midline Glioma (DMG) trials