RecruitingPHASE3INTERVENTIONAL

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

This study, called the ACTION Study, is testing a new medication named dordaviprone (ONC201) for people with a specific type of brain tumour called H3 K27M-mutant diffuse glioma. This tumour has a particular genetic change. Participants will have recently finished their standard radiotherapy treatment. Some will receive the new drug, some will receive a dummy pill (placebo) alongside the drug, and some will receive only the placebo. The main goal is to find out if dordaviprone can help patients live longer and keep the tumour from growing for a longer period. It's a large international study where neither the patients nor their doctors will know who is getting the active drug during the main part of the trial.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Jazz Pharmaceuticals

Enrolment target

510

Start

23 Jan 2023

Estimated completion

01 Jun 2028

H3 K27M Glioma

What is this study about?

This study is called the ACTION Study and it's looking into a new medication, dordaviprone (also known as ONC201), for people who have been diagnosed with a specific type of brain tumour. This tumour is called H3 K27M-mutant diffuse glioma. The 'H3 K27M-mutant' part means the tumour has a particular change in its genetic code. This study is taking place after patients have completed their usual first treatment, which is radiotherapy. The main aim is to see if adding dordaviprone to the treatment plan can help people live longer and stop the tumour from growing for a longer time.

Brain tumours are serious conditions, and researchers are always looking for better ways to treat them. This study is important because it's testing a new approach that specifically targets the genetic change found in these tumours. By using a study plan where some people get the new drug, some get the new drug with a dummy pill, and others get only a dummy pill, the researchers can fairly compare how well dordaviprone works compared to current approaches.

This is a 'Phase 3' study, which means it's one of the final stages of testing a new drug. It’s comparing dordaviprone to a placebo (a pill with no active medicine) to get clear information. The study will involve people from different countries. The results from this study will help doctors understand if dordaviprone could become a standard treatment option for H3 K27M-mutant diffuse glioma in the future.

Key takeaways

Tests a new drug (dordaviprone) for a specific brain tumour.
For people who have recently finished radiotherapy.
Aims to see if the drug extends life and slows tumour growth.
You might receive the drug or a dummy pill (placebo).
Requires regular check-ups and MRI scans.
You can stop participating at any time.

Who may be eligible?

To join this study, you need to have a specific type of brain tumour called H3 K27M-mutant diffuse glioma, which has been newly diagnosed. Doctors will check your tumour tissue to confirm this genetic change. You also need to have recently finished your radiotherapy treatment for the tumour, specifically within 2 to 6 weeks before you might join the study. Your initial radiotherapy must have started within 12 weeks of your tumour diagnosis.

You also need to be well enough to take part and weigh at least 10 kg. Your doctor will assess your overall health and ability to do daily activities. You must be willing to understand the study steps and give your written permission to participate. Also, before you started your radiotherapy, and again after you finished it, you would have needed to have good quality MRI scans of your brain.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Have I been diagnosed with H3 K27M-mutant diffuse glioma?
Did I finish my standard radiotherapy within the last 2-6 weeks?
Am I generally well enough to participate, as judged by my doctor?
Do I weigh at least 10 kilograms (about 22 pounds)?
Am I willing to have regular check-ups and MRI scans?

Answer every question to see your result.

What does participation involve?

If you join this study, you will be assigned to one of three groups: one group will receive the new drug dordaviprone, another group will receive dordaviprone along with a dummy pill (placebo), and the third group will receive only the dummy pill. The drug, or dummy pill, is taken by mouth. Neither you nor your study doctor will know which group you are in, which helps ensure the study results are fair.

Details about the number of hospital visits, how often you'll take the medication, and the total duration of your participation will be explained by the study team. You'll have regular check-ups, which will likely include physical examinations, blood tests, and MRI scans to monitor your health and the tumour. You can withdraw from the study at any time without giving a reason.

Potential risks and benefits

Potential benefits of joining include receiving a new medication that might help treat your particular type of brain tumour, and you'll be closely monitored by medical professionals. You would also be contributing valuable information that could help others with this condition in the future. However, as with any new medication, there are potential risks and side effects that are not yet fully known. The study drug may or may not be effective for you, and you might receive a placebo instead of the active drug. Your doctor will explain all known potential side effects. You are free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (162)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Banner MD Anderson Cancer Center
Verified postcode
Phoenix, United States· Completed
Barrow Neurological Institute
Verified postcode
Phoenix, United States· Active not recruiting
Phoenix Childrens Hospital
Verified postcode
Phoenix, United States· Completed
Mayo Clinic Arizona
Verified postcode
Phoenix, United States· Completed
UC San Diego Moores Cancer Center
Verified postcode
La Jolla, United States· Active not recruiting
Kaiser Permanente Los Angeles Medical Center
Verified postcode
Los Angeles, United States· Completed
UCLA University of California Los Angeles
Verified postcode
Los Angeles, United States· Completed
Children's Hospital of Orange County
Verified postcode
Orange, United States· Completed
University of California Irvine
Verified postcode
Orange, United States· Active not recruiting
UCSF Benioff Children's Hospital
Verified postcode
San Francisco, United States· Active not recruiting
University of California San Francisco
Verified postcode
San Francisco, United States· Active not recruiting
Providence Saint John's Cancer Institute
Verified postcode
Santa Monica, United States· Active not recruiting

Common questions

What is H3 K27M-mutant diffuse glioma?

It's a specific type of brain tumour that has a particular genetic change, or 'mutation', called H3 K27M.

What is dordaviprone (ONC201)?

It's the name of the new experimental drug being tested in this study to see if it can help treat this type of brain tumour.

What does 'randomised, double-blind, placebo-controlled' mean?

It means you're randomly assigned to a treatment group, and neither you nor your doctor will know if you're getting the active drug or a dummy pill (placebo). This helps make the study fair.

Why is radiotherapy mentioned?

This study is for people who have already completed their standard radiotherapy treatment for the tumour.

Can I still join if I finished radiotherapy a long time ago?

No, you must have completed your radiotherapy within 2 to 6 weeks before potentially joining the study.

How to find out more

Clinical Trial Disclosure & Transparency

clinicaltrialdisclosure@jazzpharma.com 215-832-3750 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.