Active not recruitingPHASE3INTERVENTIONAL

A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma

This research study is looking at a condition called low-grade glioma, a type of brain tumour, in children and young adults aged 2 to 21 who also have Neurofibromatosis Type 1 (NF1). The study wants to find out if a new drug called selumetinib works as well as the usual treatment, which is a combination of two chemotherapy drugs, carboplatin and vincristine. For some participants whose tumours affect their eyesight (optic pathway gliomas), the study also aims to see if selumetinib is better at improving vision. Selumetinib works by blocking certain pathways that help tumour cells grow, while the standard chemotherapy drugs stop tumour cells from dividing and spreading. This study will help doctors understand which treatment works best for patients with NF1-related low-grade glioma.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

National Cancer Institute (NCI)

Enrolment target

165

Start

15 Jan 2020

Estimated completion

31 Dec 2027

Low Grade Glioma Neurofibromatosis Type 1 Visual Pathway Glioma

What is this study about?

This study is designed for children and young adults (aged 2 to 21) who have Neurofibromatosis Type 1 (NF1) and have been diagnosed with a brain tumour called low-grade glioma. These tumours are often slow-growing, but they can cause various problems depending on their location. The main goal of this research is to compare a newer drug, selumetinib, with the current standard treatment, which involves a combination of two chemotherapy drugs: carboplatin and vincristine. Doctors want to see if selumetinib is just as good, or even better, at treating these tumours.

Selumetinib works differently from standard chemotherapy. Instead of generally attacking fast-growing cells, it focuses on specific chemical processes that low-grade glioma cells need to grow and multiply. By blocking these processes, selumetinib aims to slow or stop the tumour's growth. Carboplatin and vincristine work in other ways to kill tumour cells, stop them from dividing, or prevent them from spreading. Both treatments are given to manage the tumour.

Specifically, for patients whose tumours are affecting their vision (called optic pathway gliomas), one of the key questions the study asks is whether selumetinib might lead to better improvements in eyesight compared to the standard treatment. The study will carefully monitor tumour size, how long patients stay free of tumour growth, and overall survival, as well as looking at quality of life and other health measures. The information gathered will help doctors decide the best way to treat NF1-related low-grade gliomas in the future.

Key takeaways

This study compares a new drug (selumetinib) with standard chemotherapy for NF1-related brain tumours.
It's for children and young adults (2-21 years old) with low-grade glioma linked to NF1.
A key goal is to see if selumetinib can improve vision for those with tumours affecting eyesight.
Participation involves regular check-ups, MRI scans, eye tests, and questionnaires.
You will be randomly assigned to one of the treatments.
You can stop participating at any time.

Who may be eligible?

This study is for children and young people aged between 2 and 21 years old. To be considered, you must have Neurofibromatosis Type 1 (NF1), which can be confirmed by a doctor or through genetic tests. You also need to have a specific type of brain tumour called low-grade glioma.

Your low-grade glioma must be newly diagnosed, or if it was diagnosed before, you should not have received any treatment for it other than surgery. If your tumour is affecting your eyesight (an optic pathway glioma), you might be eligible if you have new or worsening vision problems, or if your doctor sees that the tumour is growing. If your low-grade glioma is in another part of the brain, you might be eligible if you are experiencing new or worsening symptoms, or if the tumour is growing.

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 2 and 21 years old?
Do you have Neurofibromatosis Type 1 (NF1)?
Have you been diagnosed with a low-grade glioma that hasn't been treated yet (apart from surgery)?
If your tumour affects your eyes, are you having new or worsening vision problems, or is the tumour growing?
If your tumour is elsewhere, are you having new or worsening symptoms, or is the tumour growing?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be assigned to receive either the drug selumetinib or the combination of carboplatin and vincristine. This is decided at random, like flipping a coin. You will have regular appointments for check-ups, which will include physical examinations, blood tests, and scans such as MRI (`Magnetic Resonance Imaging`) to monitor the tumour. Your vision will also be checked regularly using special eye tests.

Throughout the study, you will be asked to complete questionnaires about your quality of life, how you are feeling, and your daily activities. This helps doctors understand how the treatments affect your well-being. The total duration of your participation will depend on how you respond to the treatment, but careful follow-up will continue for a period to see the long-term effects of the treatment.

Potential risks and benefits

Participating in this study might offer a chance to receive a new treatment, selumetinib, which could potentially be as good as or better than the standard treatment, especially for vision. However, there's no guarantee of benefit, and like all medical treatments, there are potential side effects, which your doctor will explain in detail. You'll be closely monitored for any reactions. You are free to withdraw from the study at any time and for any reason, without it affecting your usual medical care.

Locations (132)

Children's Hospital of Alabama
Verified postcode
Birmingham, United States
Banner Children's at Desert
Verified postcode
Mesa, United States
Phoenix Childrens Hospital
Verified postcode
Phoenix, United States
Arkansas Children's Hospital
Verified postcode
Little Rock, United States
Loma Linda University Medical Center
Verified postcode
Loma Linda, United States
Children's Hospital Los Angeles
Verified postcode
Los Angeles, United States
Kaiser Permanente-Oakland
Verified postcode
Oakland, United States
Children's Hospital of Orange County
Verified postcode
Orange, United States
Lucile Packard Children's Hospital Stanford University
Verified postcode
Palo Alto, United States
Rady Children's Hospital - San Diego
Verified postcode
San Diego, United States
Naval Medical Center -San Diego
Verified postcode
San Diego, United States
UCSF Medical Center-Mission Bay
Verified postcode
San Francisco, United States

Common questions

What is Neurofibromatosis Type 1 (NF1)?

NF1 is a genetic condition that causes tumours to grow on nerves, usually harmless, but can sometimes cause other health problems like low-grade glioma.

What are low-grade gliomas?

Low-grade gliomas are a type of brain tumour that typically grow slowly. They are called 'low-grade' because they are less aggressive than 'high-grade' tumours.

How does selumetinib work?

Selumetinib is a drug that blocks specific signals inside the tumour cells that they need to grow. This helps to stop or slow down the tumour's growth.

What is 'standard treatment' in this study?

The standard treatment used for comparison in this study is a combination of two chemotherapy drugs called carboplatin and vincristine, which help to stop cancer cell growth.

Will I know which treatment I'm getting?

No, you won't know initially. Participants are randomly assigned to either selumetinib or the standard treatment to ensure fair comparison between the two.