AuthorisedTherapeutic exploratory (Phase II)Interventional

PNOC029_Tovorafenib for the treatment of newly diagnosed or recurrent craniopharyngioma in children and young adults

This research study, called PNOC029, is looking into a new treatment, Tovorafenib, for children and young adults who have been recently diagnosed with or have a recurring brain tumour called craniopharyngioma. This is a Phase II study, meaning we're testing how well the medicine works and if it's safe. We want to see if Tovorafenib can stop the tumour from growing for at least 12 months, which is called 'progression-free survival'. We're also very interested in understanding how this treatment affects the patients' quality of life over the same period. The aim is to find better ways to treat this condition and improve the lives of those affected.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Prinses Maxima Centrum voor Kinderoncologie B.V.

Enrolment target

Start

25 Mar 2026

craniopharyngioma

What is this study about?

This study is about a type of brain tumour called craniopharyngioma, which mostly affects children and young adults. While these tumours are usually not cancerous (they don't spread to other parts of the body), they can grow in tricky areas of the brain. This growth can cause many problems, including issues with vision, hormones, and how a person develops. Current treatments often involve surgery or radiotherapy, which can have significant side effects.

Researchers are looking for new and gentler ways to treat craniopharyngioma. This particular study is testing a new medicine called Tovorafenib. It's a 'Phase II' study, which means we're trying to find out how effective this new medicine is and if it's safe to use. The main goals are to see if Tovorafenib can stop the tumour from growing for at least 12 months and to understand how it impacts the everyday lives and well-being of the patients receiving it.

By taking part, you could be helping doctors learn more about craniopharyngioma and whether Tovorafenib could become a helpful treatment in the future. The hope is to find a treatment that is not only effective but also helps children and young adults live a better quality of life.

Key takeaways

This study tests a new medicine, Tovorafenib, for craniopharyngioma.
It's for children and young adults aged 18 and over.
The main goals are to see if the medicine stops tumour growth and improves quality of life.
You'll have regular hospital visits for health checks and eye tests.
Your participation could help improve future treatments for others.

Who may be eligible?

This study is for children and young adults who have craniopharyngioma. This includes those who have just been diagnosed or whose tumour has come back after treatment.

To join, you need to be at least 18 years old. Both males and females can take part in this study. The medical team will check other health details to make sure the study is a good fit for you.

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 18 years old or older?
Have you been diagnosed with craniopharyngioma, or has it come back?
Are you male or female?
Are you willing and able to attend regular hospital appointments?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be taking the study medicine, Tovorafenib. The exact schedule for taking the medicine would be explained by the study doctor. You would have regular visits to the hospital or clinic to see the study team. During these visits, the doctors would check your health, how you're feeling, and if the medication is working. This would involve assessments like eye tests to check your vision (including how clear your sight is and your peripheral vision), as well as tests to monitor your hormone levels. These checks help the team understand how the tumour is responding and if there are any changes to your vision or hormone levels, such as those related to growth or thirst. These checks would continue for at least 12 months and potentially for a longer follow-up period.

Potential risks and benefits

Taking part in a study like this could offer potential benefits, as Tovorafenib might help control the tumour. However, like all medicines, Tovorafenib can have side effects. The study team will explain these potential risks to you and monitor you closely for any issues. You are free to decide to leave the study at any time, for any reason, without it affecting your medical care.

Locations (3)

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

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Unverified
Netherlands
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Unverified
France
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Unverified
Germany

Common questions

What is craniopharyngioma?

It's a type of non-cancerous brain tumour that grows near an important part of the brain controlling many body functions, including vision and hormones.

What is Tovorafenib?

It's a study medicine being tested to see if it can help treat craniopharyngioma by stopping the tumour from growing.

Will I get better if I join this study?

We can't promise you'll get better, but we hope to learn if Tovorafenib can help control the tumour and improve quality of life for people with craniopharyngioma.

How long will I be in the study?

The main part of the study will involve checks for at least 12 months, with possible longer follow-up to see how you are doing over time.

What does quality of life mean in this study?

It means how the treatment affects your daily life, feelings, and overall well-being. We want to see if the medicine helps you live a better life.