RecruitingPHASE3INTERVENTIONAL

Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication 2.0

This study, called BIOMEDE 2.0, is for children and young people over 6 months old who have a type of brain tumour called Diffuse Intrinsic Pontine Glioma (DIPG) or similar tumours (Diffuse Midline Gliomas, particularly those with a specific genetic change called H3 K27M or H3K27-altered). It's a key part of a larger ongoing research programme. The main goal is to find out if a new medicine called ONC201 is more effective than another medicine called Everolimus. Participants will be randomly put into one of two groups to receive either ONC201 or Everolimus. Doctors will then carefully watch to see how well each medicine controls the tumour. If the tumour starts to grow again, participants might be able to switch to the other treatment. The findings will help decide if ONC201 could become a new standard treatment.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Gustave Roussy, Cancer Campus, Grand Paris

Enrolment target

433

Start

29 Sep 2022

Estimated completion

01 Sep 2031

Diffuse Intrinsic Pontine Glioma Diffuse Midline Glioma, H3 K27M-Mutant Diffuse Midline Glioma, H3K27-altered

What is this study about?

This study, called BIOMEDE 2.0, is designed to help children and young people over 6 months old who have a serious type of brain tumour called Diffuse Intrinsic Pontine Glioma (DIPG) or similar tumours known as Diffuse Midline Gliomas. These tumours are often very difficult to treat, and researchers are constantly looking for better options. This study focuses on tumours with a specific genetic change, which doctors call H3 K27M-Mutant or H3K27-altered, as these changes can affect how the tumour behaves and how it might respond to treatment.

The main aim of BIOMEDE 2.0 is to test and compare two different medicines: Everolimus and a new medicine called ONC201. Both medicines are taken by mouth. Participants in this study will be randomly assigned to receive one of these two treatments, meaning it's like flipping a coin for which group you join. This is a common and fair way to ensure the study results are reliable. Doctors will then closely monitor how well the medicine is working to control the tumour and manage any side effects. If a participant's tumour starts to grow, they might have the option to switch to the other medicine being tested.

The researchers hope to find out if ONC201 is significantly better at stopping the tumour from growing compared to Everolimus. The information gathered from this study, particularly how long people remain stable without the tumour growing (this is called *progression-free survival*), will help doctors understand which medicine is more effective. If ONC201 proves to be much better, it could potentially become a new standard treatment for these challenging brain tumours. The study will continue until the tumour starts to grow, side effects become too difficult to manage, or the participant decides to stop.

Key takeaways

This study is testing two medicines, Everolimus and ONC201, for specific types of aggressive brain tumours (DIPG and Diffuse Midline Gliomas).
It's for children and young people aged 6 months and older whose tumours have a specific genetic change.
Participants will be randomly assigned to receive one of the two medicines, both taken by mouth.
Doctors will closely monitor how well the treatment works and manage any side effects.
If the tumour grows, it may be possible to switch to the other study medicine.
The goal is to find out if ONC201 is a better treatment option than Everolimus.

Who may be eligible?

This study is looking for children and young people aged 6 months and older who have been diagnosed with Diffuse Intrinsic Pontine Glioma (DIPG) or a similar type of brain tumour called Diffuse Midline Glioma. It's important that these tumours have a specific genetic change which doctors refer to as H3 K27M or H3K27-altered. This genetic information helps the researchers understand if the medicines will be a good fit.

To confirm eligibility, a diagnosis of DIPG usually comes from a combination of scans and symptoms. For other Diffuse Midline Gliomas, doctors will typically confirm the diagnosis through a small sample of the tumour (a biopsy) or through surgery. In some cases, a diagnosis might be made using a 'liquid biopsy', which involves testing fluid from the spine or blood if a traditional biopsy is too risky. If a biopsy is needed, you and your family will be fully informed and asked for your consent before it happens.

Crucially, you would need to have had your diagnosis confirmed or be in the process of confirming it to ensure your tumour fits the specific type the study is investigating. The study team will review all your medical information to make sure you meet all the necessary requirements to take part safely and effectively.

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.

Are you or your child 6 months old or older?
Has a doctor diagnosed DIPG or a Diffuse Midline Glioma?
Has medical testing shown your tumour has a specific genetic change (H3 K27M or H3K27-altered)?
Are you able to take medicines by mouth?
Are you willing to have regular hospital visits, including scans and blood tests?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be assigned to one of two treatment groups, either receiving Everolimus or ONC201. This assignment is done randomly, like drawing lots. Both medicines are taken by mouth. You will have regular hospital visits for check-ups, scans, and blood tests to see how the treatment is working and to monitor for any side effects. The study treatment will continue for as long as it is helping to control your tumour and you are not experiencing severe side effects. If your tumour starts to grow again, the doctors might discuss the possibility of switching you to the other medicine being tested in the study. You will also have regular follow-up appointments to ensure your well-being after treatment stops. The full length of your participation will depend on how you respond to the treatment and your health, but you can stop at any time.

Potential risks and benefits

Taking part in a clinical trial offers the chance to access new treatments that are not yet widely available, which could potentially be more effective for your condition. The information gained from your participation will also help future patients. However, like all medicines, the treatments in this study (Everolimus and ONC201) can have side effects, and they might not work for everyone. These side effects can vary from mild to more serious, and the study team will monitor you closely to manage them. You will be given detailed information about the potential benefits and risks before you decide to join. Remember, taking part is completely voluntary, and you are free to withdraw from the study at any time without it affecting your medical care.

Locations (50)

Aarhus Universitetshospital Skejby
Verified postcode
Aarhus, Denmark· Recruiting
Rigshospitalet
Verified postcode
Copenhagen, Denmark· Recruiting
H.C. Andersen Children's Hospital, Odense Universitetshospital
Verified postcode
Odense, Denmark· Recruiting
Gustave Roussy
Verified postcode
Villejuif, France· Recruiting
CHU d'Amiens-Picardie Site Sud
Verified postcode
Amiens, France· Recruiting
Institut de Cancérologie de l'Ouest (ICO) - Site Paul Papin
Verified postcode
Angers, France· Recruiting
CHU d'Angers - Bâtiment Robert Debré
Verified postcode
Angers, France· Recruiting
CHU Besançon - Hôpital Jean Minjoz
Verified postcode
Besançon, France· Recruiting
CHU de Bordeaux - Groupe hospitalier Saint André - Hôpital Saint André
Verified postcode
Bordeaux, France· Recruiting
CHU de Bordeaux - Groupe hospitalier Pellegrin - Hôpital des enfants
Verified postcode
Bordeaux, France· Recruiting
CHRU de Brest - Hôpital Morvan
Verified postcode
Brest, France· Recruiting
CHU de Caen - Hôpital Côte de Nacre
Verified postcode
Caen, France· Recruiting

Common questions

What is DIPG and Diffuse Midline Glioma?

These are types of serious brain tumours that grow in specific parts of the brain or spinal cord. They are often difficult to treat, and this study aims to find better ways to help.

What are Everolimus and ONC201?

These are both medicines being tested in this study. Everolimus is an existing medicine, while ONC201 is a newer one that doctors hope might work better for these specific tumours.

Will I know which medicine I'm getting?

Yes, this is an 'open-label' study, meaning both you and your doctors will know which treatment you are receiving. Your progress will be reviewed by doctors who don't know which treatment you're on, to keep things fair.

What if the first medicine doesn't work for me?

If your tumour starts to grow again while on one medicine, and after careful review, you might be able to switch to the other medicine being tested in the study.

How long will I be in the study?

You will continue participating as long as the treatment is helping, your side effects are manageable, or until you choose to leave the study. There will also be follow-up appointments after treatment stops.

How to find out more

Jacques GRILL, MD, PhD

jacques.grill@gustaveroussy.fr +33 (0)1 42 11 62 09 Official trial record

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