GliomaNCT04617002
Intermediate-size Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline Gliomas
A study testing ONC201 (dordaviprone) in people with Glioma and H3 K27M, run by Jazz Pharmaceuticals at 23 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 years and older
- What is being tested?
- ONC201 (dordaviprone)
- How many people
- Not given
- Where
- 23 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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.
- Magnetic resonance imaging (MRI) of patient's glioma obtained within 28 days prior to start of ONC201 for Arms B, C, and F.
- Arm E: MRI obtained within 8 weeks prior to enrollment.
- Adequate organ and marrow function as defined below:
- Absolute neutrophil count ≥1,000/mm3 without growth factor use ≤7 days prior to treatment (Cycle 1 Day 1 [C1D1])
- Hemoglobin ≥8.0 mg/dL without red blood cell transfusion ≤3 days prior to C1D1
- Total serum bilirubin ≤1.5 X upper limit of normal (ULN) (participants with Gilbert's syndrome may be included with total bilirubin > 1.5 × ULN if direct bilirubin is ≤ 1.5 × ULN)
- AST (SGOT)/ALT (SGPT) ≤2.5 X ULN; ≤5 X ULN if there is liver involvement secondary to tumor
- Serum creatinine ≤1.5 X ULN (OR creatinine clearance ≥60 mL/min/1.73 m2) Arm E: Patients with organ and marrow function laboratory values outside the defined criteria must be approved by the medical monitor.
- Female patients of childbearing potential must agree to use an effective contraception method while taking ONC201 and for at least 90 days after the last dose of ONC201 and must have a negative pregnancy test prior to starting ONC201. Male patients must be surgically sterile (i.e., >3 months post-vasectomy) or must agree to use an effective contraception method while taking ONC201 and for at least 90 days after the last dose of ONC201. Determination of effective contraception methods will be based on the judgment of the Investigator.
- Ability to understand a written informed consent document, and the willingness to sign it. At the discretion of the Investigator, a Legally Authorized Representative (LAR) may consent on behalf of a patient who is unable to provide informed consent themselves. Assent will be obtained, as appropriate, based on the patient's age.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
23 sites in 1 country.
23 sites in 1 country
United States23 sites
Albany Medical Center
Albany, New York, 12208
University of Michigan
Ann Arbor, Michigan, 48109
University Cancer & Blood Center
Athens, Georgia, 30607
Children's Hospital of Colorado
Aurora, Colorado, 80045
Lurie Children's Hospital
Chicago, Illinois, 60661
University of Texas Southwestern
Dallas, Texas, 75390
Kapi'olani Medical Center for Women and Children
Honolulu, Hawaii, 96826
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
Kaiser Permanente Los Angeles Medical Center
Los Angeles, California, 90027
Miami Cancer Institute
Miami, Florida, 33176
New York University Langone - Active, Enrolling
New York, New York, 10016
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
University of Nebraska Medical Center - Adults Only
Omaha, Nebraska, 68198
Children's Hospital of Orange County
Orange, California, 92868
BMDACC at Banner University Medical Center Phoenix
Phoenix, Arizona, 85006
Providence Neurological Specialties Clinic
Portland, Oregon, 97225
University of Rochester
Rochester, New York, 14627
Huntsman Cancer Institute
Salt Lake City, Utah, 84112
Rady Children's Hospital
San Diego, California, 92123
Providence Saint John's Health Center
Santa Monica, California, 90404
Washington University in St. Louis
St Louis, Missouri, 63110
Overlook Medical Center/ Atlantic Health System
Summit, New Jersey, 07901
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
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.
- Being tested
- ONC201 (dordaviprone)
- Medical subject headings
- Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ONC201 (dordaviprone)Drug
ONC201 (dordaviprone) is a ClpP agonist and DRD2 antagonist.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Nov 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GliomaStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04617002
- Sponsor's protocol ID
- ONC028
- Status last verified
- Aug 2025
- First submitted
- 29 Oct 2020
- Last updated
- 20 Aug 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intermediate-size Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline Gliomas
- ClinicalTrials.gov ID
- NCT04617002
- Acronym / sponsor ID
- ONC028
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 5 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 0 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (35 listed)
- 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 -
- OR
Key exclusion criteria (9 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ONC201 (dordaviprone)ONC201 (dordaviprone) is a ClpP agonist and DRD2 antagonist. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
23 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ONC201 (dordaviprone) in people with Glioma and H3 K27M, run by Jazz Pharmaceuticals at 23 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 35 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Aug 2025.