Study of ONC201 for Patients with Newly Diagnosed H3 K27M-mutant Diffuse Glioma After Radiotherapy

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What is this study about?

This clinical trial is focused on studying a type of brain tumor called H3 K27M-mutant diffuse glioma. This is a specific kind of tumor that affects the brain and is known for having a particular genetic change, or mutation, called H3 K27M. The study is investigating a treatment using a medication known as ONC201, which is taken in the form of a capsule. The purpose of the study is to evaluate how effective ONC201 is when given to patients who have completed their initial treatment with radiation therapy.

Participants in the study will be randomly assigned to receive either the ONC201 medication or a placebo, which looks like the medication but does not contain the active ingredient. This is done to compare the effects of the actual medication against no treatment. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo, to ensure unbiased results. The study will take place over a period of time, with regular check-ups and assessments to monitor the participants’ health and the tumor’s response to the treatment.

The main goals of the study are to see if ONC201 can help improve overall survival and delay the progression of the tumor. Researchers will also look at other factors, such as any side effects experienced by participants and changes in their quality of life. The study aims to provide valuable information on whether ONC201 can be a beneficial treatment option for people with H3 K27M-mutant diffuse glioma after they have completed radiation therapy.

1 joining the study

Upon joining the study, you will be asked to provide written informed consent. This means you agree to participate in the study after understanding the procedures involved. If you are unable to provide consent, a legally authorized representative may do so on your behalf.

2 initial assessments

You will undergo an initial assessment to confirm your eligibility. This includes verifying your body weight, which must be at least 10 kg, and confirming a diagnosis of H3 K27M-mutant diffuse glioma through specific tests on tumor tissue.

You will need to have a high-quality MRI scan of your brain taken before starting radiotherapy. If you had surgery, this scan should be done after the surgery. If not, it can be done before or after a biopsy.

3 post-radiotherapy assessment

After completing standard radiotherapy, which should be done within 2 to 6 weeks before joining the study, another MRI scan of your brain will be required. This helps in assessing the effects of the radiotherapy.

4 randomization

You will be randomly assigned to receive either the study medication ONC201 or a placebo. A placebo is a substance with no active medication, used to compare the effects of the actual drug.

5 medication administration

If you are assigned to receive ONC201, you will take it in the form of a capsule. The specific dosage, frequency, and duration of administration will be provided by the study team.

6 ongoing assessments

Throughout the study, you will have regular assessments to monitor your health and the effects of the treatment. This includes checking for any side effects and measuring your overall survival and progression-free survival, which refers to the time during and after treatment that you live without the disease getting worse.

7 completion of the study

The study is expected to end by December 15, 2026. Upon completion, you will have a final assessment to evaluate the overall impact of the treatment.

Who Can Join the Study?

Able to understand the study procedures and agree to participate by providing written informed consent. This means you or your legal representative must sign a document agreeing to join the study.
Body weight must be at least 10 kg (about 22 pounds) at the time of joining the study.
Must have a new diagnosis of H3 K27M-mutant diffuse glioma. This is a specific type of brain tumor confirmed by testing the tumor tissue in a certified laboratory.
Must have at least one high-quality MRI scan of the brain with contrast before starting radiotherapy. If you had surgery to remove the tumor, this scan should be done after the surgery. If you only had a biopsy, the scan can be done before or after the biopsy.
Must have at least one high-quality MRI scan of the brain with contrast 2 to 6 weeks after finishing the initial radiotherapy. All available MRI scans done before starting the study treatment should also be provided.
Must have completed standard initial radiotherapy 2 to 6 weeks before joining the study. This radiotherapy should have been started within 12 weeks of the initial diagnosis and within 8 weeks of the most recent surgery or biopsy.
Must have a Karnofsky Performance Status or Lansky Performance Status of at least 70 at the time of joining the study. These are scales used to measure your ability to perform daily activities.
If you are taking corticosteroids (a type of medication) or anti-seizure medications, the dose must be stable or decreasing for 7 days before joining the study. A stable dose means no more than a 2 mg/day increase in the medication.
Participants can be male or female and must be within certain age ranges, which include children and adults.

Who Cannot Join the Study?

Patients who have not completed their radiotherapy treatment.
Patients with other types of brain tumors that are not H3 K27M-mutant diffuse glioma.
Patients who are not within the specified age range for the study.
Patients who are unable to follow the study procedures or instructions.
Patients who have other serious health conditions that might interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who are participating in another clinical trial at the same time.
Patients who have had a recent surgery or medical procedure that might affect the study results.
Patients who have allergies or reactions to the study medication or similar drugs.
Patients who have a history of substance abuse or addiction.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country
IRCCS Humanitas Research Hospital	Rozzano	Italy
Medical University Of Vienna	Vienna	Austria
Hospital Universitario De Salamanca	Salamanca	Spain
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Hospital Universitario Hm Sanchinarro	Madrid	Spain
Hospital Universitario De Navarra	Pamplona	Spain

Other Sites

Site Name	City	Country
Universitaetsklinikum Regensburg AöR	Regensburg	Germany
Ospedale San Raffaele S.r.l.	Milan	Italy
Universitair Medisch Centrum Utrecht	Utrecht	The Netherlands
Azienda Unita Sanitaria Locale Di Bologna	Bologna	Italy
Istituto Oncologico Veneto	Padua	Italy
Odense University Hospital	Odense	Denmark
Universitaetsklinikum Tuebingen AöR	Tuebingen	Germany
Universitaetsklinikum Mannheim GmbH	Mannheim	Germany
Aalborg University Hospital	Aalborg	Denmark
Rheinische Friedrich-Wilhelms-Universitaet Bonn	Bonn	Germany
Rigshospitalet	Copenhagen	Denmark
Fundacio De Recerca Clinic Barcelona-Institut D’Investigacions Biomediques August Pi I Sunyer	Barcelona	Spain
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Vivantes Netzwerk fuer Gesundheit GmbH	Berlin	Germany
Hospital Universitario 12 De Octubre	Madrid	Spain
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Gonpbh Uuotkbatji Fpxmkexyg	Frankfurt	Germany
Uyikikveok Hqxdypbi Cwghkju	Cologne	Germany
Eqnebxe Uttdeuaicjft Mstozjv Ckiotuu Ralnmells (gmobplj Muq	Rotterdam	The Netherlands
Apcphmm Olnlojicrie Ujgxlttamsvmn Ctcgldipbumg Dgzap Ssrpse E Dctti Scjttcn Dj Tyoxoa	Turin	Italy
Ucbdavqbdquauiykddpti Efqub Abq	Essen	Germany
Ujxmerwtdngvwvjrcmbpu Aeiksxus	Augsburg	Germany
Hipbbdzx Vfyo dxwfwvwu	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Austria	Recruiting	31.03.2023
Denmark	Recruiting	31.03.2023
Germany	Recruiting	31.03.2023
Italy	Recruiting	31.03.2023
Spain	Recruiting	31.03.2023
The Netherlands	Recruiting	31.03.2023

Trial locations

Investigated drugs:

2,4,6,7,8,9-Hexahydro-4-((2-Methylphenyl)Methyl)-7-(Phenylmethyl)Imidazo(1,2-A)Pyrido(3,4-E)Pyrimidin-5(1H)-One

ONC201 is a medication being studied for its potential to treat a type of brain tumor called H3 K27M-mutant diffuse glioma. This medication is given to patients after they have completed their radiotherapy treatment. The goal of using ONC201 is to see if it can help improve the condition of patients with this specific type of brain tumor. It works by targeting certain pathways in the cancer cells, which may help to slow down or stop the growth of the tumor. This study is designed to understand how effective ONC201 is in treating this condition compared to not using the medication. Participants in the study will receive ONC201 to see if it can provide benefits in managing their brain tumor.

H3 K27M-mutant diffuse glioma – This is a type of brain tumor that occurs primarily in children and young adults. It is characterized by a specific genetic mutation known as H3 K27M, which affects the histone proteins in cells. The disease is classified as a high-grade glioma, meaning it is aggressive and tends to grow and spread quickly within the brain. As the tumor progresses, it can cause a variety of neurological symptoms, such as headaches, seizures, and changes in behavior or cognitive function. The tumor’s growth can lead to increased pressure within the skull, further exacerbating symptoms. Over time, the disease can significantly impact the quality of life due to its effects on brain function.

Trial ID:

2022-502051-56-00

Protocol code:

ONC201-108

NCT ID:

NCT05580562

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study of ONC201 for Patients with Newly Diagnosed H3 K27M-mutant Diffuse Glioma After Radiotherapy

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?