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Looking for participantsPhase3

ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)

Sponsor: Jazz Pharmaceuticals

NCT ID: NCT05580562

View on ClinicalTrials.gov ↗

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.

H3 K27MGlioma

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

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

have a negative

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
Inclusion Criteria: 1. Able to understand the study procedures and agree to participate in the study by providing written informed consent (by participant or legally authorized representative), and assent when applicable. 2. Body weight ≥ 10 kg at time of randomization. 3. Histologically diagnosed H3 K27M-mutant diffuse glioma (new diagnosis). Detection of a missense K27M mutation in any histone H3-encoding gene detected by testing of tumor tissue (immunohistochemistry \[IHC\] or next-generation sequencing \[NGS\] in a Clinical Laboratory Improvement Amendments \[CLIA\]-certified or equivalent laboratory). \[Site to provide (as available): ≥ 11 unstained formalin-fixed paraffin-embedded (FFPE) slides from tumor tissue.\] 4. At least one, high-quality, contrast-enhanced MRI of the brain obtained prior to starting radiotherapy for submission to sponsor's imaging vendor for central read. For participants who had a surgical resection, this scan must be post-resection; for participants who did not have a resection, this scan may be pre- or post-biopsy. 5. At least one, high-quality, contrast-enhanced MRI of the brain obtained 2 to 6 weeks after completion of frontline radiotherapy. If unable to obtain contrast-enhanced imaging due to lack of venous access after multiple attempts, a patient may still be eligible after collection of a nonenhanced MRI of the brain. \[Site to also provide all available MRIs completed prior to initiating treatment with study intervention.\] 6. Received frontline radiotherapy 1. Initiated radiotherapy within 12 weeks from the initial diagnosis of H3 K27M-mutant diffuse glioma. 2. Completed radiotherapy within 2 to 6 weeks prior to randomization 3. Completed standard fractionated radiotherapy (eg. 54 to 60 Gy in 28 to 33 fractions given over approximately 6 weeks or hypofractionated radiotherapy (eg. 40 Gy in 15 fractions given over approximately 3 weeks). 7. Karnofsky Performance Status or Lansky Performance Status ≥ 70 at time of randomization. 8. Stable or decreasing dose of corticosteroids and anti-seizure medications for 7 days prior to randomization, if applicable. Stable steroid dose is defined as ≤ 2 mg/day increase (based on dexamethasone dose or equivalent dose of an alternative steroid). Exclusion Criteria: 1. Primary spinal tumor. 2. Diffuse intrinsic pontine glioma (DIPG), defined as tumors with a pontine epicenter and diffuse involvement of the pons. 3. Evidence of leptomeningeal spread of disease or cerebrospinal fluid dissemination. 4. Any known concurrent malignancy. 5. New lesion(s) outside of the radiation field. 6. Received whole-brain radiotherapy. 7. Received proton therapy for glioma. 8. Use of any of the following treatments within the specified time periods prior to randomization: 1. Dordaviprone (ONC201) or ONC206 at any time. 2. Systemic bevacizumab (includes biosimilars) at any time since the initial diagnosis of H3 K27M-mutant diffuse glioma. 3. Temozolomide within past 3 weeks. 4. Tumor treating fields at any time. 5. DRD2 antagonist within past 2 weeks. 6. Any investigational therapy within past 4 weeks. 7. Strong CYP3A4 inhibitors within 3 days. 8. Strong CYP3A4 inducers (includes enzyme-inducing antiepileptic drugs) within 2 weeks. 9. Laboratory test results meeting any of the following parameters within 2 weeks prior to randomization: 1. Absolute neutrophil count \< 1.0 × 109/L or platelets \< 75 × 109/L. 2. Total bilirubin \> 1.5 × 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). 3. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \> 2.5 × ULN. 4. Creatinine clearance ≤ 60 mL/min as calculated by the Cockcroft Gault equation (or estimated glomerular filtration rate \< 60 mL/min/1.73 m2). 10. QTc \> 480 msec (based on mean from triplicate electrocardiograms) during screening. 11. Known hypersensitivity to any excipients used in the study intervention formulation. 12. Pregnant, breastfeeding, or planning to become pregnant while receiving study intervention or within 3 months after the last dose. Participants of childbearing potential must have a negative serum pregnancy test within 72 hours prior to receiving the first dose of study intervention. 13. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection requiring systemic therapy or psychiatric illness/social situations that would limit compliance with study requirements. 14. Any other condition (eg, medical, psychiatric, or social) that, in the opinion of the investigator, may interfere with participant safety or the ability to complete the study according to the protocol.

Where Is This Study? (12 UK sites)

Addenbrooke's Hospital

Cambridge CB2 0QQ, United Kingdom

Recruiting
Site contact (verified)
Fiona Harris, MBBS, MDPrincipal Investigator
Fiona Harris, MBBS, MDf.harris3@nhs.net

Beatson West of Scotland Cancer Centre

Glasgow G12 0YN, United Kingdom

Recruiting
Site contact (verified)
Stefan Nowicki, MD, PhDPrincipal Investigator
Stefan Nowicki, MD, PhDstefan.nowicki@ggc.scot.nhs.uk

Royal Hospital for Children (Glasgow)

Glasgow G51 4TF, United Kingdom

Recruiting
Site contact (verified)
Jairam Sastry, MDPrincipal Investigator

Clatterbridge Cancer Centre - Liverpool

Liverpool L7 8YA, United Kingdom

Recruiting
Site contact (verified)
Shaveta Mehta, MD, PhDPrincipal Investigator
Shaveta Mehta, MD, PhDshaveta.mehta1@nhs.net

The Christie NHS Foundation Trust

Manchester M20 4BX, United Kingdom

Recruiting
Site contact (verified)
Catherine McBain, MDPrincipal Investigator
Catherine McBain, MDcatherine.mcbain1@nhs.net

The Royal Marsden in Sutton, Surrey

Sutton SM2 5PT, United Kingdom

Recruiting
Site contact (verified)
Fernando Carceller, MDPrincipal Investigator
Fernando Carceller, MDfernando.carceller@rmh.nhs.uk

Royal Victoria Infirmary

Newcastle upon Tyne NE1 4LP, United Kingdom

Recruiting
Site contact (verified)
Joanne Lewis, MRCPCHPrincipal Investigator
Joanne Lewis, MRCPCHjoanne.lewis@nhs.net

Freeman Hospital

Newcastle upon Tyne NE7 7DN, United Kingdom

Recruiting
Site contact (verified)
Joanne Lewis, MRCPCHPrincipal Investigator
Joanne Lewis, MRCPCHjoanne.lewis@nhs.net

St James University Hospital

Leeds LS9 7TF, United Kingdom

Recruiting
Site contact (verified)
Fiona CollinsonPrincipal Investigator

The Leeds Teaching Hospitals NHS Trust, Leeds General Infimary

Leeds LS1 3EX, United Kingdom

Recruiting
Site contact (verified)
Simone Wilkins, MDPrincipal Investigator
Simone Wilkins, MDsimonewilkins@nhs.net

Guy's Hospital

London SE19RT, United Kingdom

Recruiting
Site contact (verified)
Lucy Brazil, MDPrincipal Investigator

Churchill Hospital

Oxford Ox3 7LE, United Kingdom

Recruiting
Site contact (verified)
Meera Nandhabalan, MDPrincipal Investigator
Meera Nandhabalan, MDmeera.nandhabalan@ouh.nhs.uk

How to Get in Touch

Clinical Trial Disclosure & Transparency

Sponsor contact

CONTACT

215-832-3750 ClinicalTrialDisclosure@jazzpharma.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-04