Intermediate-size Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline Gliomas
1 other identifier
expanded_access
N/A
1 country
23
Brief Summary
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.
Trial Health
Trial Health Score
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23 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 29, 2020
CompletedFirst Posted
Study publicly available on registry
November 5, 2020
CompletedAugust 20, 2025
August 1, 2025
October 29, 2020
August 15, 2025
Conditions
Interventions
ONC201 (dordaviprone) is a ClpP agonist and DRD2 antagonist.
Eligibility Criteria
You may qualify if:
- Patient meets one or more of the criteria below:
- Arm A - Closed to further enrollment.
- Arm B - Diffuse intrinsic pontine glioma (DIPG) defined as tumors with a pontine epicenter and diffuse involvement of the pons (at the discretion of the Medical Monitor, the site may be requested to provide the most recent imaging report(s) to confirm diagnosis). H3 K27M status does not have to be known or positive for this arm.
- Arm C -
- Patients with primary spinal glioma that is positive for the H3 K27M mutation (performed in a laboratory with Clinical Laboratory Improvement Amendments \[CLIA\] or equivalent certification). Primary spinal glioma must be documented in radiology reporting.
- Patients with diffuse glioma that is positive for the H3 K27M mutation (performed in a laboratory with CLIA or equivalent certification) AND radiographic evidence of leptomeningeal disease. Leptomeningeal disease must be documented in radiology reporting.
- Arm D - Closed to further enrollment.
- Arm E - Patients with H3 K27M-mutant glioma or a midline glioma of unknown H3 K27M mutational status who received ONC201 and/or ONC206 from an alternative (non-Chimerix) source prior to 31 December 2023, as evidenced by supporting documentation (e.g., medical records, pharmacy receipts, etc.). Other supporting documentation may be confirmed by the medical monitor. Detection of H3 K27M mutation should be performed in a CLIA-certified or equivalent laboratory.
- Arm F - Patients with H3 K27M-mutant diffuse glioma who have progressed during or after completion of frontline radiotherapy. Detection of H3 K27M mutation should be performed in a CLIA-certified or equivalent laboratory. Enrollment in this arm will be individually evaluated by the Sponsor and requires at least 3 days for review.
- Disease status:
- Arm B - Patient is not required to have radiographic or clinical evidence of progressive disease.
- Arm C - Patient must have progressive disease as defined by Response Assessment in Neuro-Oncology (RANO) criteria or have documented recurrent glioma on diagnostic biopsy.
- Arm E - Not applicable.
- Arm F - Patient must have progressive disease as defined by RANO criteria or have documented recurrent glioma on diagnostic biopsy.
- Prior radiotherapy:
- +19 more criteria
You may not qualify if:
- Qualifies for participation in an ongoing ONC201 or ONC206 clinical trial.
- Arms B, C, and F: Previous or current enrollment in an ONC201 or ONC206 clinical study (including open-label and blinded studies) or expanded access protocol or previous exposure to ONC201 from any source for the treatment of CNS tumor.
- Arm E: Previous or current enrollment in an ONC201 clinical study (including open label and blinded studies) or expanded access protocol for the treatment of CNS tumor.
- Current or planned participation in a study of an investigational agent (including ONC206) or using an investigational device.
- (Not applicable; criterion removed in Version 4).
- Any known systemic infection that, in the opinion of the Investigator, could compromise the safety of the patient while taking ONC201.
- Prolongation of QT/QTcF interval (QTc interval \>480 milliseconds) using Fridericia's QT correction formula on two electrocardiograms (ECGs) separated by at least 2 days.
- A history of Torsades de pointes or heart failure, hypokalemia, or family history of prolonged QT Syndrome.
- Concomitant use of medication(s) known to prolong the QT/QTc interval.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (23)
BMDACC at Banner University Medical Center Phoenix
Phoenix, Arizona, 85006, United States
Kaiser Permanente Los Angeles Medical Center
Los Angeles, California, 90027, United States
Children's Hospital of Orange County
Orange, California, 92868, United States
Rady Children's Hospital
San Diego, California, 92123, United States
Providence Saint John's Health Center
Santa Monica, California, 90404, United States
Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
Miami Cancer Institute
Miami, Florida, 33176, United States
University Cancer & Blood Center
Athens, Georgia, 30607, United States
Kapi'olani Medical Center for Women and Children
Honolulu, Hawaii, 96826, United States
Lurie Children's Hospital
Chicago, Illinois, 60661, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
Washington University in St. Louis
St Louis, Missouri, 63110, United States
University of Nebraska Medical Center - Adults Only
Omaha, Nebraska, 68198, United States
Overlook Medical Center/ Atlantic Health System
Summit, New Jersey, 07901, United States
Albany Medical Center
Albany, New York, 12208, United States
New York University Langone - Active, Enrolling
New York, New York, 10016, United States
University of Rochester
Rochester, New York, 14627, United States
Providence Neurological Specialties Clinic
Portland, Oregon, 97225, United States
University of Texas Southwestern
Dallas, Texas, 75390, United States
Huntsman Cancer Institute
Salt Lake City, Utah, 84112, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 29, 2020
First Posted
November 5, 2020
Last Updated
August 20, 2025
Record last verified: 2025-08