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

APPROVED_FOR_MARKETING

Conditions studied: Glioma, H3 K27M

In brief

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.

Key facts

Study ID

NCT04617002

Run by

Jazz Pharmaceuticals

Last updated by the study team

2025-08-20

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

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.
• OR
• 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:
• Arm B - Patient must be at least 14 days from completion of radiotherapy.
• Arm C - Patient must be at least 30 days from completion of frontline radiotherapy and at least 14 days from reirradiation if applicable.
• Arm E - Not applicable.
• Arm F - Patient must be at least 30 days from completion of frontline radiotherapy and at least 14 days from reirradiation if applicable.
• (Not applicable; criterion removed in Version 5).
• Patients must weigh at least 10 kg.
• Washouts:
• Arms B, C, and F - From the projected start of scheduled study treatment, the following time periods must have elapsed from prior anti-cancer treatments: 5 half-lives from any investigational agent, 4 weeks from cytotoxic therapy (except 23 days for temozolomide and 6 weeks from nitrosoureas), 6 weeks from anti-cancer antibodies (no washout required for bevacizumab), 4 weeks (or 5 half-lives, whichever is shorter) from other anti-tumor therapies including CAR-T and other cellular therapies/immunotherapies, and 1 week from devices used to treat cancer.
• Arm E - No washouts are required for ONC201 and ONC206. All other anti-cancer agents need to be discontinued prior to enrollment with the exception of bevacizumab.

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.

Where it is running

• BMDACC at Banner University Medical Center Phoenix — Phoenix, Arizona, United States
• Kaiser Permanente Los Angeles Medical Center — Los Angeles, California, United States
• Children's Hospital of Orange County — Orange, California, United States
• Rady Children's Hospital — San Diego, California, United States
• Providence Saint John's Health Center — Santa Monica, California, United States
• Children's Hospital of Colorado — Aurora, Colorado, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• Miami Cancer Institute — Miami, Florida, United States
• University Cancer & Blood Center — Athens, Georgia, United States
• Kapi'olani Medical Center for Women and Children — Honolulu, Hawaii, United States
• Lurie Children's Hospital — Chicago, Illinois, United States
• University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
• University of Michigan — Ann Arbor, Michigan, United States
• Washington University in St. Louis — St Louis, Missouri, United States
• University of Nebraska Medical Center - Adults Only — Omaha, Nebraska, United States
• Overlook Medical Center/ Atlantic Health System — Summit, New Jersey, United States
• Albany Medical Center — Albany, New York, United States
• New York University Langone - Active, Enrolling — New York, New York, United States
• University of Rochester — Rochester, New York, United States
• Providence Neurological Specialties Clinic — Portland, Oregon, United States
• University of Texas Southwestern — Dallas, Texas, United States
• Huntsman Cancer Institute — Salt Lake City, Utah, United States
• Children's Hospital of The King's Daughters — Norfolk, Virginia, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.