Active not recruitingPHASE2INTERVENTIONAL

Trametinib for Pediatric Neuro-oncology Patients With Refractory Tumor and Activation of the MAPK/ERK Pathway.

This study is for children and young people aged 1 month to 25 years who have certain types of brain tumours (low-grade glioma) or nerve tumours (plexiform neurofibroma) that have not improved with usual treatments. It is testing a medicine called trametinib to see how well it works. This is a "Phase 2" study, meaning researchers want to find out if the medicine is safe and effective in this group of patients. The study will also explore how these tumours grow and why some might not respond to treatment. An important part of the study includes looking at how treatment affects the patient's quality of life and brain function. Around 150 patients will take part.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

St. Justine's Hospital

Enrolment target

114

Start

16 Aug 2018

Estimated completion

01 Mar 2027

Low-grade Glioma Plexiform Neurofibroma Central Nervous System Glioma

What is this study about?

This study is looking into a medicine called trametinib for children and young people who have specific types of brain tumours, known as low-grade glioma, or a type of nerve tumour called plexiform neurofibroma. These conditions often need special attention because they can be challenging to treat. For most patients joining, their tumour will have already been treated with other medicines like chemotherapy or radiotherapy, but these haven't worked well enough or the tumour has come back. For patients with plexiform neurofibroma, this might be their first treatment study as there isn’t a standard treatment for it yet.

The main goal of this study is to see how effectively trametinib shrinks these tumours or stops them from growing. Researchers are also very keen to learn more about the biology of these tumours – what makes them grow, why some treatments work better than others, and what causes them to become resistant to treatment. This knowledge can help develop better treatments in the future.

Beyond just looking at tumour size, this study also focuses on what really matters to patients and their families. It includes checking on their quality of life, which means how they feel day-to-day, and doing tests to see how the treatment might affect memory, thinking, and learning. This comprehensive approach helps us understand the full picture of how trametinib might impact young patients.

Key takeaways

This study is for children and young people (1 month to 25 years) with certain brain or nerve tumours.
It tests a medicine called trametinib, usually for tumours that haven't responded to previous treatments.
The study aims to see if trametinib works and to understand more about these tumours.
It will also check how the treatment affects quality of life and brain function.
Around 150 patients will take part, with daily medicine for up to 18 cycles (28 days each).

Who may be eligible?

This study is for children and young people aged between 1 month (after their due date if they were born early) and 25 years old. To join, you would generally have one of the following conditions: a low-grade brain tumour (glioma) linked to a condition called neurofibromatosis type 1 (NF1), a nerve tumour called plexiform neurofibroma also linked to NF1, a low-grade brain tumour with a specific genetic change (called BRAF fusion), or any type of brain tumour that shows activity in a particular cell pathway (MAPK/ERK pathway).

For most types of tumours in this study, you must have tried at least one other treatment, like chemotherapy or radiation, that wasn't successful. However, if you have plexiform neurofibroma, you might not have needed previous treatment. Also, any side effects from your past treatments should have mostly cleared up.

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 between 1 month (corrected age) and 25 years old?
Do you have a low-grade brain tumour (glioma) often with certain gene changes, or a plexiform neurofibroma linked to NF1?
For most tumour types, have you tried at least one other treatment that wasn't successful?
Are any past treatment side effects mostly gone or mild?
Are you able to attend regular hospital visits for check-ups and scans?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be given the study medicine, trametinib, as a liquid or tablet by mouth every day. You'll continue taking this medicine for up to 18 cycles, with each cycle lasting 28 days. This means the treatment period could be quite long, possibly over a year, depending on how you respond.

Throughout the study, you'll have regular visits to the hospital for check-ups. These visits will involve blood tests, physical examinations, and scans (using contrast-enhanced MRI) to see how the tumour is responding. The study will also involve tests to check your memory, thinking, and learning abilities (neuropsychological evaluations) and surveys to understand your quality of life. Before you start, the medical team will need a sample of your tumour tissue, often from a previous biopsy or surgery. They will also need to review previous MRI scans.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit if trametinib helps shrink your tumour or stops its growth, especially if other treatments haven't worked well. However, there's no guarantee the medicine will help, and it might have side effects, just like any medication. These will be fully explained to you before you decide to join. You are completely free to withdraw from the study at any time, for any reason, and this will not affect your future medical care.

Locations (7)

Alberta Children's Hospital
Verified postcode
Calgary, Canada
Children and Women's Health Centre of British Colombia
Verified postcode
Vancouver, Canada
IWK Health Centre
Verified postcode
Halifax, Canada
The Hospital for Sick Children
Verified postcode
Toronto, Canada
CHU Sainte-Justine
Verified postcode
Montreal, Canada
Montreal Children's Hospital
Verified postcode
Montreal, Canada
CHU de Québec
Verified postcode
Québec, Canada

Common questions

What is trametinib?

Trametinib is a medicine that targets specific pathways within cells to slow down the growth of cancer cells.

What does 'low-grade glioma' mean?

Low-grade glioma is a type of brain tumour that usually grows slowly and is less aggressive than high-grade tumours.

What is 'Phase 2' in a clinical trial?

Phase 2 means the study is testing if a new medicine is safe and effective for a specific group of patients, after initial safety tests.

Will I have to pay to be in the study?

No, you will not have to pay for the study medicine or the extra tests related to being part of the study.

Can I stop being in the study if I change my mind?

Yes, you can decide to leave the study at any time, and it will not affect your regular medical care.