AuthorisedTherapeutic exploratory (Phase II)Interventional

A randomized phase II study comparing the safety and efficacy of mifamurtide and standard therapy containing sorafenib in pediatric and young adult patients with high-risk osteosarcoma

This study is for children and young adults with a type of bone cancer called osteosarcoma, specifically when it's high-risk, has returned, or hasn't responded to previous treatments. It compares a new medicine called mifamurtide, given along with standard treatment (which includes sorafenib), to standard treatment alone. The main goal is to see how long patients live without their cancer growing or coming back, which doctors call "event-free survival." Researchers will also check overall survival, how many people's tumours shrink, and any side effects. This is a Phase II study, meaning it's an earlier stage of research looking for signs of effectiveness and safety.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Instytut Matki I Dziecka

Enrolment target

Start

20 Apr 2026

Relapsed/refractory osteosarcoma, High-grade osteosarcoma

What is this study about?

This research study is looking at a type of bone cancer called osteosarcoma. Specifically, it's for young people whose osteosarcoma is considered high-risk, has come back after treatment, or hasn't responded well to previous treatments. Doctors are trying to find better ways to help these patients. The study is comparing two approaches: one group will receive standard treatment that includes a medicine called sorafenib, and another group will get the same standard treatment but with an additional medicine called mifamurtide. Mifamurtide is being investigated to see if it can improve how well the standard treatment works.

The main thing the doctors want to find out is how long patients live without their cancer getting worse, returning, or without passing away. This is called 'event-free survival'. They will also be looking at overall survival (how long people live in total), how many people's tumours shrink or disappear, and carefully checking for any side effects or feelings of unwellness that might occur with these treatments. This is a very important part of making sure treatments are not only effective but also safe.

The trial is in 'Phase II', which means it's an important step after initial safety tests. The researchers are trying to see if there's enough promise and benefit from adding mifamurtide to standard therapy to justify larger studies in the future. The information gathered from this study will help healthcare professionals understand if this new combination could be a good option for certain patients with osteosarcoma.

Key takeaways

This study is for young people with high-risk or difficult-to-treat osteosarcoma.
It compares standard treatment with standard treatment plus a new medicine called mifamurtide.
The main goal is to see if the new combination can help patients live longer without their cancer returning or getting worse.
Researchers will also carefully watch for any side effects of the treatments.
Participation involves regular hospital visits, tests, and closely following the treatment plan.

Who may be eligible?

This study is looking for children and young adults who have a specific type of bone cancer called osteosarcoma. This includes patients whose cancer is considered high-risk, has come back after treatment, or hasn't responded well to previous medicines.

To be considered for the study, you must be 18 years old or older. The study is open to both male and female patients.

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.

I have been diagnosed with osteosarcoma that is high-risk, has come back, or is hard to treat.
I am 18 years old or older.
I am open to receiving standard treatment or standard treatment plus a new investigational medicine.
I am able to attend regular hospital appointments and follow the study schedule.

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be randomly assigned to receive one of two treatment plans. This means you wouldn't get to choose which treatment you receive; it's like flipping a coin to ensure fairness in comparing the treatments. Both groups will involve receiving a medicine called sorafenib, and one group will also receive mifamurtide. You will have regular hospital visits for check-ups, blood tests, and scans (like X-rays or MRI scans) to see how you are responding to the treatment and to keep an eye on your general health.

These visits and tests will help the doctors understand how well the treatments are working and if there are any side effects. You will need to take the study medicines as prescribed. The total length of your participation, including follow-up appointments after your main treatment, will be discussed in detail by the study team, as it can vary depending on individual response and the study's schedule.

Potential risks and benefits

Taking part in a clinical trial offers potential benefits, such as access to new treatments like mifamurtide that might be more effective than current standard care for your specific type of osteosarcoma. You'll also receive very close medical attention and monitoring. However, there are potential risks, including side effects from the medications, which can vary from mild to severe, and the possibility that the treatment may not work for you. The study team will explain all known side effects and monitor you carefully for any new ones. Remember, your participation is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without affecting your future medical care.

Locations (1)

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

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Unverified
Poland

Common questions

What is osteosarcoma?

Osteosarcoma is a type of cancer that starts in the bones. It mostly affects children and young adults.

What does 'high-risk' osteosarcoma mean?

High-risk means the cancer might be more likely to spread or come back, making it harder to treat successfully.

What are sorafenib and mifamurtide?

These are medicines used in cancer treatment. Sorafenib is a standard treatment, and mifamurtide is being tested to see if it can improve outcomes when added to standard care.

What does 'randomized' mean in a study?

It means you'd be put into one treatment group or another by chance, like flipping a coin, to make sure the comparison is fair.

Can I stop participating in the study if I want to?

Yes, absolutely. You can withdraw from the study at any time without having to give a reason, and it won't affect your future medical care.