Actively Recruiting
ONC206 for Treatment of Newly Diagnosed, Recurrent Diffuse Midline Gliomas, and Other Recurrent Malignant CNS Tumors
Led by Sabine Mueller, MD, PhD · Updated on 2026-03-17
208
Participants Needed
6
Research Sites
309 weeks
Total Duration
On this page
Sponsors
S
Sabine Mueller, MD, PhD
Lead Sponsor
J
Jazz Pharmaceuticals
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
ONC206 for Treatment of Newly Diagnosed, Recurrent Diffuse Midline Gliomas, and Other Recurrent Malignant CNS Tumors
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Children and young adults aged 2 to 21 years with DMG, H3K27 altered, who have completed at least one prior therapy including focal radiation (Arm A).
- Newly diagnosed children and young adults aged 2 and older with DMG, H3K27 altered, including spinal cord DMGs (Arm B).
- Children and young adults aged 2 to 21 years with progressing DMG, H3K27 altered, who have not yet been treated for progression and are candidates for re-irradiation (Arm C).
- Children and young adults aged 2 to 21 years with recurrent primary malignant CNS tumors, excluding DMGs, with progression and no prior treatment for progression (Arm D).
- Tumor tissue confirmation consistent with diagnosis is required.
- Participants must have recovered from acute side effects of prior therapy.
- Specific washout periods from prior therapies must be met before starting study treatment.
- Participants must be on stable or decreasing steroid doses for at least 3 days before registration.
- Adequate blood counts and organ function tests within specified limits.
- Well-controlled seizure disorder allowed.
- Agreement to use adequate contraception during study participation for females of child-bearing potential and males.
- Karnofsky or Lansky performance score of 50 or higher.
- Willingness to provide adequate tumor tissue.
- Ability to understand and sign informed consent/assent.
- Enrollment on PNOC COMP if available.
You will not qualify if you...
- Patients with thalamic or cerebellar DMG H3K27 altered without concurrent therapy other than temozolomide (Arms A & B).
- Prior participation in ONC201 trials in the upfront setting (Arms C & D).
- Current use of other investigational or anti-cancer drugs.
- Known immune system disorders requiring systemic immunosuppressive therapy.
- Uncontrolled infections.
- Pregnant or breastfeeding females; positive pregnancy test before therapy.
- Active illicit drug use or alcoholism.
- History of allergic reactions to similar compounds as ONC206.
- Inability to follow study procedures due to language, psychological, or cognitive issues.
- Illnesses affecting ONC206 absorption.
- Use of strong CYP3A4, 2D6, 1A2, 2C9, or 2C19 inhibitors or inducers within 14 days prior and during study.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 6 locations
1
University of California, San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
2
Emory University
Atlanta, Georgia, United States, 30322
Actively Recruiting
3
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
4
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
5
Princess Máxima Center for Pediatric Oncology
Utrecht, Netherlands, 3584
Actively Recruiting
6
The University Children's Hospital in Zurich
Zurich, Canton of Zurich, Switzerland, 8032
Actively Recruiting
Research Team
P
PNOC Operations
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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