At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Dordaviprone (ONC201) (drug), Dordaviprone (ONC201) + Placebo (drug), Placebo (other)
- How long the study runs
- Study runs about 65 months (dates as stated)
- About the drug or intervention
- Dordaviprone (ONC201) — drug: Participants ≥ 52.5 kg will receive 625 mg of dordaviprone (5 × 125-mg capsules) dosing days; participants \< 52.5 kg will receive a dose (and corresponding number of capsules) scaled by body weight and rounded to 125-mg increments. · Dordaviprone (ONC201) + Placebo — drug: Participants ≥ 52.5 kg will receive 625 mg of dordaviprone (5 × 125-mg capsules) or matching placebo on dosing days; participants \< 52.5 kg will receive a dose (and corresponding number of capsules) scaled by body weight and rounded to 125-mg increments · Placebo — other: Participants will receive placebo (same number of capsules as the dordaviprone dose) on dosing days
- Patient visit burden
- Not specified by the sponsor
In plain English
The ACTION study, run by Jazz Pharmaceuticals, is testing a drug called ONC201 (also known as dordaviprone) in people newly diagnosed with a type of brain tumour called H3 K27M-mutant diffuse glioma, after they have finished radiotherapy. H3 K27M is a specific change (mutation) found in the tumour. Not stated — ask the trial team.
Who can take part
- Able to understand the study and agree to take part, with written consent (a legally authorised representative can consent, and children may give assent where applicable)
- Body weight of at least 10 kg when joining the study
- Newly diagnosed diffuse glioma with an H3 K27M mutation, confirmed by testing the tumour tissue
- At least one high-quality MRI brain scan with contrast dye taken before radiotherapy started
- At least one high-quality MRI brain scan with contrast dye taken 2 to 6 weeks after finishing radiotherapy (a scan without contrast may be accepted if contrast is not possible after several attempts)
- Radiotherapy started within 12 weeks of diagnosis and finished 2 to 6 weeks before joining the study, using a standard schedule (for example 54 to 60 Gy over about 6 weeks) or a shorter schedule (for example 40 Gy over about 3 weeks)
- Karnofsky or Lansky Performance Status score of at least 70 (a measure of how well you can carry out daily activities)
- On a stable or reducing dose of steroid and anti-seizure medicines for the 7 days before joining the study, if taking them (stable means no more than a 2 mg per day increase in dexamethasone or an equivalent steroid)
Who may not be able to
- Tumour started in the spine
- Diffuse intrinsic pontine glioma (DIPG), meaning a tumour centred in and spread through the pons area of the brainstem
- Evidence the cancer has spread to the linings of the brain or spinal cord, or into the spinal fluid
- Another cancer at the same time
- New tumours outside the area treated with radiotherapy
- Having had whole-brain radiotherapy or proton therapy for the glioma
- Previous treatment with ONC201 (dordaviprone) or ONC206, or tumour treating fields, at any time
- Bevacizumab (including biosimilars) at any time since diagnosis
- Temozolomide within the past 3 weeks
- A DRD2 antagonist medicine within the past 2 weeks
- Any experimental (investigational) therapy within the past 4 weeks
- Strong CYP3A4 inhibitor medicines within the past 3 days, or strong CYP3A4 inducer medicines (including some anti-seizure drugs) within the past 2 weeks
- Blood, liver or kidney test results below or above certain limits within 2 weeks of joining (for example, low white blood cells or platelets, raised liver enzymes, or reduced kidney function)
- A heart tracing (electrocardiogram) showing a QTc over 480 milliseconds at screening
- Known allergy to any of the other ingredients in the study drug
- Pregnant, breastfeeding, or planning to become pregnant during the study or within 3 months after the last dose. Anyone who could become pregnant needs a negative pregnancy blood test within 72 hours before the first dose
- An uncontrolled other illness, such as an infection needing treatment, or a mental health or social situation that would make it hard to follow the study requirements
- Any other condition the study doctor believes could affect safety or ability to complete the study
What taking part involves
- • Taking the study drug ONC201 (dordaviprone) after finishing radiotherapy. Not stated — ask the trial team about how it is taken, the dose, or the length of treatment
- • Providing tumour tissue samples, including at least 11 unstained slides if available
- • Having MRI brain scans, including scans before and 2 to 6 weeks after radiotherapy, and sharing all scans taken before starting study treatment
Time commitment: Not stated — ask the trial team about the number of visits, how long the study lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- Not specified
- Who
- All
- Number of participants
- 510
- Started
- 2023-01-23
- Last checked
- 2026-04
Plain English Summary
What is this study?
- • Testing a new treatment for h3 k27m
- • Phase3 - 510 participants
- • This is a randomized, double-blind, placebo-controlled, parallel-group, international, Phase 3 study in patients with newly diagnosed H3 K27M-mutant diffuse glioma to assess whether treatment with dordaviprone (ONC201) following frontline radiotherapy will extend overall survival and progression-free survival in this population
Who can take part?
- • Adults
- • Diagnosed with h3 k27m
Where?
- • Cambridge - Addenbrooke's Hospital
- • Glasgow - Beatson West of Scotland Cancer Centre
- • Glasgow - Royal Hospital for Children (Glasgow)
- • Liverpool - Clatterbridge Cancer Centre - Liverpool
- • +8 more UK sites
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is a randomized, double-blind, placebo-controlled, parallel-group, international, Phase 3 study in patients with newly diagnosed H3 K27M-mutant diffuse glioma to assess whether treatment with dordaviprone (ONC201) following frontline radiotherapy will extend overall survival and progression-free survival in this population. Eligible participants will have histologically diagnosed H3 K27M-mutant diffuse glioma and have completed standard frontline radiotherapy.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: Not specified
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Able to understand the study procedures and agree to participate in the study by providing written agreement to take part...
- ✓Body weight ≥ 10 kg at time of randomization.
- ✓Received frontline radiotherapy
- ✓Initiated radiotherapy within 12 weeks from the initial diagnosis of H3 K27M-mutant diffuse glioma.
- ✓Completed radiotherapy within 2 to 6 weeks prior to randomization
Who cannot take part
- ✗Primary spinal tumor.
- ✗Diffuse intrinsic pontine glioma (DIPG), defined as tumors with a pontine epicenter and diffuse involvement of the pons.
- ✗Evidence of leptomeningeal spread of disease or cerebrospinal fluid dissemination.
- ✗Any known concurrent cancer.
- ✗New lesion(s) outside of the radiation field.
See the full criteria
Where Is This Study? (12 UK sites)
Addenbrooke's Hospital
Cambridge CB2 0QQ, United Kingdom
Beatson West of Scotland Cancer Centre
Glasgow G12 0YN, United Kingdom
Royal Hospital for Children (Glasgow)
Glasgow G51 4TF, United Kingdom
Clatterbridge Cancer Centre - Liverpool
Liverpool L7 8YA, United Kingdom
The Christie NHS Foundation Trust
Manchester M20 4BX, United Kingdom
The Royal Marsden in Sutton, Surrey
Sutton SM2 5PT, United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne NE1 4LP, United Kingdom
Freeman Hospital
Newcastle upon Tyne NE7 7DN, United Kingdom
St James University Hospital
Leeds LS9 7TF, United Kingdom
The Leeds Teaching Hospitals NHS Trust, Leeds General Infimary
Leeds LS1 3EX, United Kingdom
Guy's Hospital
London SE19RT, United Kingdom
Churchill Hospital
Oxford Ox3 7LE, United Kingdom
How to Get in Touch
Clinical Trial Disclosure & Transparency
Sponsor contactCONTACT
