Ongoing, recruitingPhase I and Phase II (Integrated)- OtherInterventional

A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in Pediatric Patients with Solid Tumors Dependent on KIT or PDGFRA Signaling

This research study is investigating a new medication called Avapritinib for children and young people who have solid tumours where the cancer's growth is linked to specific signals within their cells (called KIT or PDGFRA). This is a two-part study. The first part focuses on finding the safest and most effective dose of Avapritinib for this age group, while also carefully watching for any side effects. The second part will then use this determined dose to see how well the medicine works at shrinking or controlling the cancer. We'll also look at how the body handles the medicine and continue to monitor for any side effects. The goal is to provide valuable information about Avapritinib as a potential treatment option for these particular cancers in young patients.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Blueprint Medicines Corp.

Enrolment target

Start

12 May 2024

Solid Tumors Dependent on KIT or PDGFRA Signaling

What is this study about?

This study is about a new medicine called Avapritinib, which we hope could help children and young people with certain types of solid tumours. Solid tumours are growths of abnormal cells in the body, and in this case, the cancers being studied are those that depend on specific signals (called KIT or PDGFRA) to grow. These signals are like instructions that tell the cancer cells to keep multiplying.

The study has two main goals. The first is to find the best dose of Avapritinib for young patients. This involves giving different small doses to see which one works without causing too many side effects. We want to find a dose that is strong enough to potentially help but also safe enough for children and young people. We'll be carefully monitoring participants for any side effects throughout this process.

The second main goal, once the right dose is found, is to see how well Avapritinib actually works against these cancers. This means checking if the medicine can make the tumours shrink or stop growing. We'll also be looking at how the medicine moves through the body and how long it stays there. The information gathered from this study is really important for understanding if Avapritinib could be a useful new treatment option in the future for young patients with these specific types of cancer.

Key takeaways

It's an early-stage study (Phase 1/2) for a new cancer medicine.
The medicine is called Avapritinib, for children with specific solid tumours.
The study aims to find a safe dose and see how well it works.
It's for cancers reliant on KIT or PDGFRA signals.
Participation involves regular hospital visits for checks and tests.

Who may be eligible?

This study is open to children and young people of all ages and genders. You would be considered for this study if you have a solid tumour that your doctors believe is growing because of specific signals (called KIT or PDGFRA) in the cancer cells. Your doctors will be able to tell you if your type of cancer fits this description.

There will be other specific medical checks and tests that the study doctors will need to carry out to make sure it's safe for you to take part and that the study is the right fit for your situation. These checks are important to protect your health.

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.

Do I have a solid tumour?
Has my doctor said my cancer has KIT or PDGFRA signals?
Am I a child or young person (any age)?
Am I able to attend regular hospital appointments?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would receive the study medicine, Avapritinib. The study has two parts. In the first part, we'll be trying to find the best dose, so the amount of medicine you receive might change slightly over time, but always under careful medical supervision. In the second part, once the best dose is found, you would receive that specific dose.

Taking part would involve regular visits to the hospital for doctors’ appointments, blood tests, and scans (like MRI or CT scans) to check how you’re doing, how the medicine is working, and to look for any side effects. You might also be asked to complete some simple questionnaires about how you feel, especially about how you like taking the medicine. The total duration of your participation would depend on how you respond to the treatment and what the doctors decide is best for you, but you would be kept informed every step of the way.

Potential risks and benefits

Participating in this study might offer the potential benefit of accessing a new treatment (Avapritinib) that is not yet widely available, which could possibly help control or shrink your cancer. However, as with any new medicine, there are potential risks, including side effects that we don't fully know yet or that could be serious. All possible side effects will be explained to you and carefully monitored throughout the study. You will always have the right to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

—
Unverified
Austria
—
Unverified
France
—
Unverified
Germany
—
Unverified
Italy

Common questions

What is Avapritinib?

Avapritinib is a new medicine being studied to see if it can treat certain types of cancer by targeting specific growth signals within the cancer cells.

Will I get better if I join this study?

We hope the medicine will help, but we cannot guarantee that it will make you better or cure your cancer. This study is to find out if it can help.

What are 'solid tumours'?

Solid tumours are lumps or growths of abnormal cells in the body, such as those found in organs or tissues.

Will I have to stay in the hospital?

You will have regular hospital visits for check-ups and tests, but it's not expected that you would stay in the hospital for the entire study unless medically necessary.

Who is paying for this study?

The study is sponsored by a pharmaceutical company, but this does not affect your medical care or the study results.