GliomaNCT03134131
Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline High Grade Gliomas
A study testing ONC201 in people with Glioma, run by Jazz Pharmaceuticals at 1 site in United States.
Trial summary
The objective of this expanded access program is to provide ONC201 to eligible patients with previously-treated glioma that exhibits the H3 K27M mutation and/or that is located in the midline region of the brain. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Glioma
- What is being tested?
- ONC201
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 1 siteUnited 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 3 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Patient must have one type of diagnosis below:
- A glioma that is positive for the H3 K27M mutation (performed in a laboratory with CLIA certification);
- A grade III or IV glioma involving the thalamus, hypothalamus, brainstem, cerebellum, midbrain, or spinal cord;
- Diffuse intrinsic pontine glioma (DIPG), defined as tumors with a pontine epicenter and diffuse involvement of the pons. These patients are eligible with or without a tissue biopsy.
- Unequivocal evidence of progressive disease on as defined by RANO criteria or have documented recurrent glioma on diagnostic biopsy.
- Patient must have had previous therapy that includes radiotherapy.
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- Interval of at least 90 days from the completion of radiotherapy to the first dose of ONC201. If patients are within 90 days of radiotherapy, they may still be eligible if they meet one or more of the following criteria.
- Progressive tumor is outside the original high-dose radiotherapy target volume as determined by the treating investigator, or
- Histologic confirmation of tumor through biopsy or resection, or
- Nuclear medicine imaging, MR spectroscopy, or MR perfusion imaging consistent with true progressive disease, rather than pseudoprogression or radiation necrosis obtained within 28 days of registration.
- Patient must be at least 3 years of age.
- Patient must weigh at least 10kg.
- Patient must be able to swallow and retain orally administered medication. For patients unable to swallow capsules, oral ONC201 will be administered as a liquid formulation in Ora-Sweet.
- 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 (except 21 days for bevacizumab), or 4 weeks (or 5 half-lives, whichever is shorter) from other anti-tumor therapies.
- Contrast-enhanced head CT or brain MRI within 21 days prior to start of study drug.
- 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)
- AST (SGOT)/ALT (SGPT) ≤2 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)
- For patients post pubertal: Female patients must agree to use effective contraception while taking ONC201 and for at least 90 days after completion of treatment. Male patients must be surgically sterile or must agree to use effective contraception while taking ONC201 and for at least 90 days after completion of treatment. The decision of effective contraception will be based on the judgment of the principal investigator.
- Ability to understand a written informed consent document, and the willingness to sign it. Assent will be obtained when appropriate based on the subjects age.
Who cannot join
- Qualifies for participation in an ongoing ONC201 clinical trial or is already participating in an ONC201 clinical trial.
- Current or planned participation in a study of an investigational agent or using an investigational device.
- Evidence of diffuse leptomeningeal disease or CSF dissemination.
- Any known systemic infection that, in the opinion of the investigator, could compromise the safety of the patient, while taking ONC201.
Trial locations and contacts
1 site in 1 country.
About this study
The objective of this expanded access program is to provide ONC201 to eligible patients with previously-treated glioma that exhibits the H3 K27M mutation and/or that is located in the midline region of the brain.
- Conditions
- Glioma
- Being tested
- ONC201
- Medical subject headings
- Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ONC201Drug
ONC201 is an oral, small molecule selective antagonist of DRD2 that induces tumor cell death.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Apr 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- NCT03134131
- Sponsor's protocol ID
- ONC018
- Status last verified
- Oct 2020
- First submitted
- 20 Apr 2017
- Last updated
- 14 Mar 2022
- Results posted
- No
Publications
Arrillaga-Romany I, Gardner SL, Odia Y, Aguilera D, Allen JE, Batchelor T, Butowski N, Chen C, Cloughesy T, Cluster A, de Groot J, Dixit KS, Graber JJ, Haggiagi AM, Harrison RA, Kheradpour A, Kilburn LB, Kurz SC, Lu G, MacDonald TJ, Mehta M, Melemed AS, Nghiemphu PL, Ramage SC, Shonka N, Sumrall A, Tarapore RS, Taylor L, Umemura Y, Wen PY. ONC201 (Dordaviprone) in Recurrent H3 K27M-Mutant Diffuse Midline Glioma. J Clin Oncol. 2024 May 1;42(13):1542-1552. doi: 10.1200/JCO.23.01134. Epub 2024 Feb 9.
PubMed 38335473
Study at a glance
From the registry record, last updated by the sponsor on 14 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline High Grade Gliomas
- ClinicalTrials.gov ID
- NCT03134131
- Acronym / sponsor ID
- ONC018
- Conditions
- Glioma
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 28 Apr 2017 (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.
The objective of this expanded access program is to provide ONC201 to eligible patients with previously-treated glioma that exhibits the H3 K27M mutation and/or that is located in the midline region of the brain.
Study design
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (23 listed)
- Patient must have one type of diagnosis below:
- Unequivocal evidence of progressive disease on as defined by RANO criteria or have documented recurrent glioma on diagnostic biopsy.
- Patient must have had previous therapy that includes radiotherapy.
- Interval of at least 90 days from the completion of radiotherapy to the first dose of ONC201. If patients are within 90 days of radiotherapy, they may still be eligible if they meet one or more of the following criteria.
- Patient must be at least 3 years of age.
Key exclusion criteria (4 listed)
- Qualifies for participation in an ongoing ONC201 clinical trial or is already participating in an ONC201 clinical trial.
- Current or planned participation in a study of an investigational agent or using an investigational device.
- Evidence of diffuse leptomeningeal disease or CSF dissemination.
- Any known systemic infection that, in the opinion of the investigator, could compromise the safety of the patient, while taking ONC201.
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 |
|---|---|---|
| ONC201ONC201 is an oral, small molecule selective antagonist of DRD2 that induces tumor cell death. | 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 14 Mar 2022.
- Derived from this study: PubMed 38335473Arrillaga-Romany I, Gardner SL, Odia Y, Aguilera D, Allen JE, Batchelor T, Butowski N, Chen C, Cloughesy T, Cluster A, de Groot J, Dixit KS, Graber JJ, Haggiagi AM, Harrison RA, Kheradpour A, Kilburn LB, Kurz SC, Lu G, MacDonald TJ, Mehta M, Melemed AS, Nghiemphu PL, Ramage SC, Shonka N, Sumrall A, Tarapore RS, Taylor L, Umemura Y, Wen PY. ONC201 (Dordaviprone) in Recurrent H3 K27M-Mutant Diffuse Midline Glioma. J Clin Oncol. 2024 May 1;42(13):1542-1552. doi: 10.1200/JCO.23.01134. Epub 2024 Feb 9.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ONC201 in people with Glioma, run by Jazz Pharmaceuticals at 1 site 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 23 inclusion and 4 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 14 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
On the ClinicalTrials.gov record for NCT03134131, which also lists 1 linked publication.
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 14 Mar 2022.