Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

An open randomized phase II clinical trial evaluating the safety and efficacy of rapamycin in the treatment of gliomas high-grade malignant gliomas in children as part of the establishment management of rare and ultra rare diseases of the central nervous system associated with mTOR pathway activation: BraimTOR- ONKO

This study is looking at a medicine called Rapamune (rapamycin) for children diagnosed with high-grade glioma, which is a serious type of brain tumour. The main goal is to find out how safe the medicine is by carefully checking for any side effects, especially serious ones. Doctors also want to see if Rapamune can help children live longer, prevent the tumour from growing, or make it shrink. This is a "Phase II" trial, meaning it's an important step to see if a new treatment works well enough to be studied in a larger group of people. The information from this study will help improve treatments for rare brain conditions where certain body processes (called the mTOR pathway) are overactive.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Instytut Pomnik Centrum Zdrowia Dziecka

Enrolment target

Start

11 Jan 2025

high grade glioma

What is this study about?

This study is about a medicine called Rapamune, which contains rapamycin. Doctors are giving this medicine to children who have a type of brain tumour known as high-grade glioma. These tumours are quite serious, and the aim of this study is to see if Rapamune can be a useful treatment.

The main thing doctors want to learn first is how safe Rapamune is. They will be looking very closely at any side effects that children might experience while taking the medicine and for some time afterwards. They will also be checking to see if the medicine helps children live longer, if it stops the tumour from growing, or if it makes the tumour shrink. This helps them understand how well the treatment is working.

This study is also part of a larger effort to find better treatments for rare conditions that affect the brain and spinal cord, especially those linked to a specific pathway in the body called the 'mTOR pathway'. By understanding how Rapamune works in these particular tumours, researchers hope to develop more targeted and effective therapies for children with these challenging diseases.

Key takeaways

Tests Rapamune for children with a serious type of brain tumour (high-grade glioma).
Focuses on safety and side effects of the treatment.
Also aims to see if the medicine helps the tumour or improves long-term health.
A "Phase II" study, an important step in finding new treatments.
Aims to improve care for rare brain conditions.
Open to children of all ages, both boys and girls, with specific tumour types.

Who may be eligible?

This study is for children of any age who have been diagnosed with a high-grade glioma, which is a serious type of brain tumour. Both boys and girls can take part.

Before joining, doctors will need to check if Rapamune is suitable for your child, as other health conditions or previous treatments might mean they can't take part. They will explain all the details thoroughly to make sure it's the right fit for your child.

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.

Has my child been diagnosed with a high-grade glioma (a serious brain tumour)?
Has my child's doctor suggested this study might be suitable?
Am I comfortable with my child taking a new medicine being tested in a study?
Can my child attend regular check-ups and follow-up appointments?
Have I discussed all other treatment options with my child's medical team?

Answer every question to see your result.

What does participation involve?

If your child joins this study, they will take the Rapamune medicine as an oral solution (a liquid by mouth). Throughout the study, doctors will keep a very close eye on your child to see how the medicine is affecting them. This will involve regular check-ups, which might include scans and blood tests, to look for any side effects and to see how the tumour is responding. The doctors will also track your child's health for a long time after the treatment phase, specifically at 15 months, 24 months, 3 years, and 5 years, to understand the long-term effects of the medicine.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. One potential benefit is that the study medicine, Rapamune, might help to treat your child's brain tumour, perhaps by shrinking it or slowing its growth. However, there's no guarantee it will work for every child, and it might not provide any direct benefits. Like all medicines, Rapamune can have side effects, and the study will be carefully monitoring for these to understand them better. You and your child always have the right to withdraw from the study at any time, for any reason, without it affecting your child's usual medical care.

Locations (1)

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

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Unverified
Poland

Common questions

What is a high-grade glioma?

It's a serious type of brain tumour in children that grows quickly. Finding new ways to treat it is very important.

What is Rapamune (rapamycin)?

It's a medicine being tested in this study to see if it can help treat these brain tumours. It comes as a liquid you take by mouth.

What does 'Phase II' mean?

It means this is an early stage of testing. Doctors are looking closely at how safe the medicine is and if it shows promise before doing larger studies.

Will my child get the study medicine?

Yes, in this type of study, all participants who are eligible will receive the study medicine, Rapamune.

What if we change our minds about participating?

You and your child can choose to stop participating in the study at any time, and it will not affect your child's medical care.