Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options

This research study, called a clinical trial, is looking into a new medicine called Entrectinib. It's designed for children and teenagers (up to 18 years old) who have solid tumours (growths in the body) or tumours in the brain. These tumours are either advanced, have spread to other parts of the body, or standard treatments haven't been successful for them. The study aims to find out the best dose of Entrectinib to give and to see how well it works against these types of tumours, especially those with specific genetic changes called NTRK1/2/3 or ROS1 gene fusions. This trial offers a potential new treatment option for those with limited choices.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Hoffmann-La Roche

Enrolment target

Start

03 May 2016

Estimated completion

30 Jun 2026

Solid Tumors CNS Tumors

What is this study about?

This study is a research project exploring a new medicine called Entrectinib for children and young people. We're looking for those aged up to 18 years who have solid tumours (growths in the body) or brain tumours that are quite advanced, have spread, or for which current treatments haven't worked well or aren't suitable. The main goal is to find out if Entrectinib is a safe and effective treatment option for these young patients.

The study is divided into two main parts. The first part, which has now finished, focused on finding the safest dose of Entrectinib for children with various solid tumours. The second part, which is ongoing, is looking more closely at how well Entrectinib works, especially in patients whose tumours have particular genetic changes, known as NTRK1/2/3 or ROS1 gene fusions. These genetic changes can sometimes make tumours grow, and Entrectinib is designed to target them.

By taking part in this study, young patients might get access to a new treatment that isn't widely available yet. The information we gather will help doctors understand more about Entrectinib and whether it could become a standard treatment in the future for children and teenagers facing these challenging cancers. We hope this research will lead to better treatment options for young people in the UK and worldwide.

Key takeaways

This study is for children and teenagers (up to 18 years old) with advanced solid or brain tumours.
It's testing a new medicine called Entrectinib.
Entrectinib targets specific genetic changes (NTRK1/2/3 or ROS1 gene fusions) in tumours.
It offers a potential new treatment option when standard treatments are not suitable or haven't worked.
Participation involves regular hospital visits, tests, and taking the study medicine.
You can withdraw your child from the study at any time.

Who may be eligible?

This study is for children and teenagers up to 18 years old with certain types of cancer. To be considered, your child's tumour must be either a solid tumour (a growth found outside the brain) or a brain tumour. For many parts of the study, we'll need to see how big the tumour is and if it's changing, using scans or other tests.

A key part of eligibility is that your child's cancer is advanced, has spread, or that standard treatments haven't worked or aren't good options. For some parts of the study, it's very important that the tumour has specific genetic changes called NTRK1/2/3 or ROS1 gene fusions. This means we'll need to test the tumour tissue to see if these changes are present.

Your child also needs to be well enough to take part. This is assessed by their doctor, who will look at their general health and how well they can do daily activities. They should also have recovered from any recent treatments and be expected to live for at least four more weeks.

Quick self-check

Is your child aged 18 or under?
Does your child have a solid tumour outside the brain or a brain tumour that is advanced or has spread?
Do other treatments no longer work well, or are they not suitable for your child's condition?
Is your child well enough to participate in a study, as assessed by their doctor?
Does your child's tumour have specific genetic changes called NTRK1/2/3 or ROS1 gene fusions? (This is important for some parts of the study.)

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

What does participation involve?

If your child takes part in this study, they will receive the study medicine, Entrectinib. The exact schedule for hospital visits, tests, and how long your child will receive the medicine will be explained by the study team. Typically, this involves regular check-ups, blood tests, and scans (like MRI or CT scans) to monitor their health and how their tumour is responding to the treatment. The total duration of participation will vary for each child, depending on how they respond to the treatment and their overall health. The study team will provide a detailed plan specific to your child's situation. There will also be follow-up appointments after stopping the medication to ensure their well-being.

Potential risks and benefits

Taking part in any clinical trial comes with potential benefits and risks. A potential benefit of this study is that your child might receive a new treatment, Entrectinib, that could help their tumour when other options haven't worked. However, like all medicines, Entrectinib can have side effects, which the study team will explain to you in detail. There will also be extra hospital visits and tests compared to standard treatment. It's important to remember that you have the right to withdraw your child from the study at any time, for any reason, without it affecting their future medical care.

Locations (26)

University of California San Diego
La Jolla, United States
UCSF Benioff Children's Hospital
San Francisco, United States
Children's Hospital Colorado
Aurora, United States
Egleston Children's Hospital at Emory University Atlanta
Atlanta, United States
University of Chicago
Chicago, United States
Johns Hopkins University
Baltimore, United States
Dana Farber Cancer Institute
Boston, United States
Washington University,St. Louis Children's Hospital
St Louis, United States
Memorial Sloan Kettering Cancer Center
New York, United States
Nationwide Children's Hospital
Columbus, United States
Oregon Health & Science Uni
Portland, United States
Children's Hospital of Philadelphia
Philadelphia, United States

+14 more sites — see the official record for the full list.

Common questions

What is Entrectinib?

Entrectinib is the name of the new medicine being tested in this study. It's designed to target specific genetic changes that can occur in some cancers.

What are NTRK1/2/3 or ROS1 gene fusions?

These are specific genetic changes that can happen in the DNA of some cancer cells. Entrectinib is designed to block the signals from these altered genes, which might help stop the cancer from growing.

Will my child definitely get the new medicine?

Yes, in this study, all participants who are eligible and join will receive Entrectinib.

How long will my child need to be in the study?

The length of time varies for each child, depending on how they respond to the treatment and their overall health. The study team will discuss the expected duration with you.

What if we change our mind about participating?

You are free to withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.