RecruitingPhase I/IIInterventional

5G: A next generation agile genomically guided glioma modular platform for proof-of-concept molecular hypothesis testing in patients with malignant brain tumours

This study, called 5G-PEARL, is looking into a new drug called paxalisib alongside temozolomide, a common treatment for glioblastoma (a serious brain tumour). Paxalisib works by blocking a pathway in cancer cells that helps them grow. Researchers want to find out if this combination treatment is safe and if it can shrink tumours or help patients. They are particularly interested in patients whose tumours have a specific feature that makes them more likely to respond to paxalisib. The study will check for side effects and how well the treatment works. It's a Phase I/II study, meaning it's still early stage, focusing on both safety and initial signs of effectiveness.

At a glance

Status

Recruiting

Phase

Phase I/II

Sponsor

Institute of Cancer Research

Enrolment target

182

Start

13 Feb 2026

Estimated completion

13 Feb 2029

Glioblastoma

What is this study about?

The 5G-PEARL study is a clinical trial that aims to find better ways to treat a type of brain tumour called glioblastoma. This study is testing a new drug named paxalisib, which works by targeting a specific pathway inside cancer cells that helps them grow and spread. Think of this pathway like a busy road that cancer cells use a lot; paxalisib tries to block this road to slow them down. Paxalisib is being studied for other types of cancer too, and is now being looked at for brain tumours.

In this study, paxalisib is given together with temozolomide (TMZ). TMZ is a standard chemotherapy drug that works by damaging the cancer cells' DNA – their genetic instruction manual – preventing them from growing and dividing. By combining these two drugs, researchers hope to achieve a stronger effect against the glioblastoma cells. The main goal of this early phase of the study is to see if this combination is safe for patients and if there are any signs that it helps to shrink the tumour or improve the patient's condition.

Researchers are especially interested in selecting patients whose brain tumours have a particular feature – an "overactive PI3K signalling pathway." This is because paxalisib is designed to block this specific pathway, so patients with this feature might be more likely to benefit from the treatment. This approach helps to make the study more targeted, focusing the new drug on those who might gain the most from it.

Key takeaways

This study is testing a new drug (paxalisib) for glioblastoma.
It combines paxalisib with a standard treatment (temozolomide).
The main goals are to check for safety and signs of effectiveness.
Patients whose tumours might respond best to paxalisib are preferred.
Participation involves regular check-ups and extra scans.
You can withdraw from the study at any time.

Who may be eligible?

To be able to join this study, people generally need to be 16 years old or older. You would need to have been diagnosed with a specific type of glioblastoma (glioblastoma, IDH-wildtype Grade 4) and have had surgery and standard radiation and chemotherapy treatments already.

You would also need to be reasonably fit and well for someone with your condition, meaning you can mostly look after yourself. Your doctor would check your blood tests to make sure your body is working properly, for example, your blood counts, liver, and kidney function. You should also be stable neurologically, meaning your brain-related symptoms haven't recently gotten worse.

Before you can take part, you would need to agree to join the Minderoo Precision Brain Tumour Programme, which involves checking the genetic makeup of your tumour. This helps researchers understand if your tumour has the specific features that might respond best to the new drug. If you are female and could become pregnant, you would need a negative pregnancy test and agree to use effective contraception during and for some time after the study.

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 16 years old or older?
Do you have a specific type of glioblastoma (IDH-wildtype Grade 4)?
Have you already had surgery and standard radiation/chemotherapy?
Are you generally well and able to mostly look after yourself?
Are you willing to use effective contraception during and after the study if needed?
Are you willing to provide samples for genetic testing of your tumour?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive the new drug, paxalisib, alongside temozolomide (TMZ). These medications would be given according to a specific schedule decided by the study doctors. You would have regular check-ups, which include visits to the clinic, blood tests, and physical examinations by the study doctor and research nurses. These are important for monitoring your health and checking for any side effects.

You would also have more scans than usual for your condition, such as CT, MRI, and potentially bone scans. These scans help doctors see how the tumour is responding to the treatment. The study is expected to run from July 2023 to August 2029, and your participation would involve treatment and follow-up during this period. The exact length of your participation would depend on how you respond to the treatment and the study plan.

Throughout the study, you would be closely monitored. If you are a woman who could become pregnant, or a man whose partner could become pregnant, you would need to use two forms of effective contraception during the study and for six months after your last dose of the study drug. All pregnancies that occur during this time must be reported to the study team.

Potential risks and benefits

It's important to know that because paxalisib is a new drug, we don't yet know if it will definitely help everyone who takes it. You might not experience any direct health benefit from taking part. The potential benefits are still being investigated. As with many cancer treatments, there's a risk of serious side effects, and some effects might not be known yet. The study team will carefully discuss all possible risks with you and monitor you closely with regular checks, blood tests, and physical exams to ensure your safety. Taking part also means having more scans (CT, MRI) which involve some exposure to radiation or magnetic fields, and potential discomfort. You always have the right to withdraw from the study at any time if you change your mind, and you will be informed promptly of any new information or side effects that emerge.

Locations (3)

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

The Royal Marsden Hospital – Drug Development Unit
City only
Sutton, England
University Hospital Southampton NHS Foundation Trust
City only
Southampton, England
Edinburgh Cancer Centre, Western General Hospital
City only
Edinburgh, Scotland

Common questions

What is glioblastoma?

Glioblastoma is a serious type of brain tumour. This study is trying to find new ways to treat it.

What is paxalisib?

Paxalisib is a new drug being tested. It works by blocking a specific pathway in cancer cells that helps them grow, aiming to slow down or stop the tumour.

What does 'Phase I/II' mean?

This means the study is in an early stage. Phase I primarily checks if the treatment is safe, and Phase II looks for initial signs of how well it works.

Will I have more scans than usual?

Yes, you would have extra CT, MRI, and possibly bone scans to help doctors monitor your tumour's response to the treatment.

Can I leave the study if I want to?

Yes, you are free to withdraw from the study at any time, for any reason, without it affecting your usual care.

How to find out more

- 5G Team

5G-enquiries@icr.ac.uk +44 203 437 6003 Official trial record

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