RecruitingPHASE1, PHASE2INTERVENTIONAL

5G-RUBY: Avutometinib and Defactinib in Malignant Brain Tumours

This research trial, called 5G-RUBY, is looking into new treatments for aggressive brain tumours such as glioblastoma. The study will test two new medications, called avutometinib and defactinib, to see how safe they are and if they can help shrink tumours. Sometimes, temozolomide, a known chemotherapy drug, might also be used alongside these new drugs. The trial focuses on patients whose tumours have certain genetic changes. It will take place in two main stages: first, focusing on safety and initial signs of effectiveness, and then, if successful, moving on to further test how well the treatments work. The goal is to find better ways to treat these challenging brain cancers.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Institute of Cancer Research, United Kingdom

Enrolment target

182

Start

15 Nov 2024

Estimated completion

30 Sep 2030

Glioblastoma Multiforme (GBM)Glioblastoma Multiform (Grade IV Astrocytoma)Diffuse Hemispheric Glioma, H3 G34-Mutant Malignant Primary Gliomas

What is this study about?

This study, called 5G-RUBY, is a research trial designed to investigate new ways to treat certain types of aggressive brain tumours, including glioblastoma, which is the most common and serious form of brain cancer. The main goal is to test two new drugs, avutometinib and defactinib, to see if they are safe for patients and if they can help to slow down or even shrink these tumours. The research also wants to understand if these new drugs work better for patients whose tumours have specific genetic changes.

Brain tumours are very complex, and treatments often need to be tailored to each person. This study is part of a larger project called Minderoo 5G, which aims to use detailed genetic information about a person's tumour to guide treatment. By looking at specific genetic changes, researchers hope to find treatments that are more likely to work for certain patients.

The trial will happen in two stages. The first stage will carefully check the safety of the new drugs and look for early signs that they might be helping. If these early results are promising, the study will move to a second stage to more thoroughly evaluate how effective the treatments are. Sometimes, the new drugs might be given on their own, or sometimes they might be combined with a standard chemotherapy drug called temozolomide, depending on what the research shows.

Key takeaways

New drugs (avutometinib, defactinib) being tested for aggressive brain tumours.
Aims to check safety and if tumours shrink.
Focuses on patients with specific genetic changes in their tumour.
May involve combination with existing chemotherapy (temozolomide).
Participation includes taking study drugs and regular medical checks.
Patients can withdraw from the study at any time.

Who may be eligible?

To be considered for the first part of this study, you would generally need to be diagnosed with a specific type of advanced brain tumour, such as certain glioblastoma or diffuse hemispheric glioma types, and your doctors will need to have confirmed this with a tissue sample.

Your tumour would also need to have specific genetic changes identified through special tests. If your tumour has returned after previous treatments (like surgery and chemotherapy), or if you are receiving treatment for minimal remaining disease after your initial surgery and chemotherapy, you might be eligible. You need to be at least 16 years old, generally well with a good quality of life, and able to look after yourself.

There are also specific requirements for your general health, such as blood test results showing your body is working well enough to participate. You must also be able to understand and agree to take part in the study by signing a consent form. The research team will be able to tell you more about the specific health checks required.

Quick self-check

Are you 16 years old or older?
Do you have a diagnosis of glioblastoma or a similar high-grade brain tumour?
Has your doctor confirmed that your tumour has specific genetic changes?
Are you able to take medicines by mouth and attend regular hospital visits?
Are you generally well and able to manage daily activities?
Are you able to give your informed consent to participate?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you would take two new drugs: avutometinib and defactinib. Avutometinib is taken by mouth at a dose of 3.2mg twice a week (for example, on Mondays and Thursdays) and can be taken with or without food. Defactinib is also taken by mouth, but at a dose of 200mg twice a day, and you'd need to take it within 30 minutes after eating.

You would have regular visits to the hospital for checks and tests to monitor your health and see how the treatment is affecting you and your tumour. These visits would involve blood tests, scans, and discussions with the study team. The total duration of your participation would depend on how your treatment is progressing and the overall study plan. Initially, the study will involve 12 patients in each group and if the early results are promising, it may progress to a larger group in the second phase.

Potential risks and benefits

Participating in a clinical trial may offer potential benefits, such as access to new treatments that are not yet widely available and close medical monitoring. However, there are also risks, as these are new drugs and their side effects are not fully known, though they will be carefully monitored. There's no guarantee the treatment will work for you. You are always free to withdraw from the study at any time, for any reason, without affecting your future medical care.

Locations (3)

Cambridge University Hospitals
Cambridge, United Kingdom· Recruiting
The Royal Marsden Hospital - Drug Development Unit
Sutton, United Kingdom· Recruiting
The Royal Marsden Hospital - Neuro-Oncology Unit
Sutton, United Kingdom· Recruiting

Common questions

What types of brain tumours is this study looking at?

This study is mainly for people with aggressive brain tumours like glioblastoma (high-grade type) and diffuse hemispheric glioma, especially those with certain genetic changes.

What are avutometinib and defactinib?

These are two new experimental drugs being tested in this study to see if they can help treat these types of brain tumours.

Will I also get standard treatments?

Depending on the study stage and your situation, you might receive the new drugs alone, or in combination with temozolomide, which is a standard chemotherapy drug.

Who can join this study?

You need to be at least 16 years old, have a specific type of brain tumour with certain genetic changes, and meet other health criteria. Your doctor can help check if you are eligible.

What if the treatment doesn't work for me?

You'll be closely monitored throughout the study. If the treatment isn't helping or if you experience significant side effects, the study team will discuss your options and you can withdraw at any time.

How to find out more

5G Team

5G-enquiries@icr.ac.uk +44 (0)2034376003 Official trial record

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