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RecruitingPHASE2INTERVENTIONAL

A Study to Evaluate Tovorafenib in Pediatric and Young Adult Participants With Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors

This study, called a Phase 2 trial, is testing a new medicine called Tovorafenib. It's for children and young adults, from 6 months to 25 years old, in the UK who have specific types of brain tumours (low-grade glioma) or certain advanced solid tumours. These tumours must have either returned or gotten worse after previous treatments and must show particular genetic changes. The study aims to see how safe Tovorafenib is and how well it works. Before joining, doctors will check if participants have the right genetic changes in their tumours. The study involves a screening period, receiving the medicine, and then follow-up checks over time.

At a glance

Status
Recruiting
Phase
PHASE2
Sponsor
Day One Biopharmaceuticals, Inc.
Enrolment target
141
Start
22 Apr 2021
Estimated completion
31 May 2027

What is this study about?

This research study is investigating a new medication called Tovorafenib. It's designed for children and young adults, aged between 6 months and 25 years, who have specific health conditions. These conditions are certain types of brain tumours, called low-grade gliomas, or other advanced solid tumours. The study is particularly focused on those whose tumours have come back or gotten worse despite previous treatments.

The main goal of this study is to understand two important things about Tovorafenib: first, how safe it is for young people, and second, how effective it is in treating their tumours. Researchers are especially interested in tumours that have a particular genetic change, which they will check for before anyone joins the study. This kind of genetic test is standard practice and helps ensure the medicine is being tested on the most suitable patients.

This is a Phase 2 study, which means it builds on earlier research that has already shown some promise. These types of studies are crucial steps in finding new and better ways to treat serious conditions, and they help doctors learn more about potential new medicines.

Key takeaways

  • This study is for children and young adults (6 months to 25 years old).
  • It's testing a new medicine called Tovorafenib.
  • It's for specific brain tumours (low-grade glioma) or advanced solid tumours.
  • Tumours must have returned or gotten worse and have specific genetic changes.
  • The aim is to check the safety and effectiveness of the new medicine.
  • Participation involves screening, treatment, and follow-up appointments.

Who may be eligible?

For this study, they are looking for children and young adults between 6 months and 25 years old. People who might be able to join have either a specific type of brain tumour called low-grade glioma, or an advanced solid tumour. For both conditions, the tumour must have either returned or gotten worse after other treatments.

Crucially, the doctors need to find specific genetic changes within the tumour cells. This is a very important part of deciding if someone can join the study, as the medicine is designed to work best when these changes are present. Participants must also have had at least one previous treatment for their cancer that didn't fully work.

However, there are also reasons someone might not be able to join. For example, if their tumour has other known genetic changes that could interfere with the study, or if they have a condition called neurofibromatosis type 1 (NF-1). The medical team will carefully review each person's health to see if the study is a good fit.

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.

  1. Are you between 6 months and 25 years old?
  2. Do you have a low-grade glioma or an advanced solid tumour?
  3. Has your tumour come back or gotten worse after previous treatments?
  4. Does your tumour have a specific genetic change (doctors will check this)?
  5. Have you had at least one prior cancer treatment?
  6. Do you NOT have a known diagnosis of neurofibromatosis type 1 (NF-1)?
Answer every question to see your result.

What does participation involve?

If you consider joining this study, it would involve several stages. First, there's a 'screening period' where doctors will do tests, including looking for specific genetic changes in your tumour. If you qualify and decide to take part, you'll then move into the 'treatment period' where you will receive the study medicine, Tovorafenib. You'll have regular appointments for check-ups and to see how you're responding to the treatment. This will involve appointments at the hospital or clinic.

After the main treatment period, some participants may enter a 'long-term extension phase', meaning they might continue to receive the medicine if it's helping. Once treatment stops, there will be 'end of treatment' visits, followed by safety check-ups to monitor your well-being. Finally, there will be 'long-term follow-up assessments' to keep an eye on your health over a longer period. The total duration of your participation would depend on how you respond to the treatment and the study's specific plan, but it will involve ongoing check-ups for some time.

Potential risks and benefits

Participating in a clinical trial like this could offer potential benefits, such as accessing a new treatment like Tovorafenib before it's widely available. It also contributes to medical knowledge that could help others in the future. However, there are also potential risks; new medicines can have side effects that are not yet fully known or can affect people differently. The study team will monitor you closely for any unwanted effects. It's important to remember that joining is entirely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (35)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • UCSF Benioff Children's Hospital
    Verified postcode
    San Francisco, United States· Recruiting
  • Children's National Medical Center
    Verified postcode
    Washington D.C., United States· Recruiting
  • Lurie Children's Hospital of Chicago
    Verified postcode
    Chicago, United States· Recruiting
  • Johns Hopkins Hospital
    Verified postcode
    Baltimore, United States· Terminated
  • Dana-Farber Cancer Institute
    Verified postcode
    Boston, United States· Recruiting
  • CS Mott Children's Hospital
    Verified postcode
    Ann Arbor, United States· Recruiting
  • St. Louis Children's Hospital
    Verified postcode
    St Louis, United States· Recruiting
  • NYU Langone Health
    Verified postcode
    New York, United States· Recruiting
  • Duke Cancer Center
    Verified postcode
    Durham, United States· Recruiting
  • Doernbecher Children's Hospital Oregon & Health Science University
    Verified postcode
    Portland, United States· Terminated
  • Children's Hospital of Philadelphia
    Verified postcode
    Philadelphia, United States· Recruiting
  • Texas Children's Hospital
    Verified postcode
    Houston, United States· Recruiting

Common questions

What is Tovorafenib?

Tovorafenib is the new medicine being tested in this study. It's a type of drug designed to target specific changes found in some cancer cells.

What is a 'low-grade glioma'?

A low-grade glioma is a type of brain tumour that tends to grow slowly. This study is for children and young adults where this type of tumour has come back or gotten worse.

What does 'advanced solid tumour' mean?

This refers to other types of cancers in the body (not blood cancers) that have spread or are at a later stage, and have also come back or progressed.

Why do you need to check for genetic changes?

The medicine, Tovorafenib, is designed to work against specific genetic changes in tumour cells. Checking for these ensures the medicine is given to those it's most likely to help.

Can I stop participating if I want to?

Yes, you can choose to leave the study at any time, for any reason. Your decision will not affect your usual medical care.

How to find out more

Day One Biopharmaceuticals, Inc.

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

Interested in taking part?

Register your interest

Share your details and the research team for "A Study to Evaluate Tovorafenib in Pediatric and Young Adult…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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