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A study comparing mifamurtide to sorafenib for treating high-risk osteosarcoma in children and young adults

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on patients with osteosarcoma, which is a type of bone cancer. The research specifically looks at individuals with high-grade osteosarcoma, a more aggressive form of the disease, or cases that are relapsed/refractory, meaning the cancer has returned after previous treatment or has not responded to initial therapy. The purpose of the study is to compare the effectiveness and safety of a new treatment against the current standard care.

Participants will be assigned to receive either mifamurtide, which is administered through intravenous use (delivered directly into a vein), or a standard treatment containing sorafenib, which is taken as an oral use medication. The study will monitor how well the treatments work over time and track any side effects that may occur during the course of the therapy.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    <b>randomization</b>

    A process occurs where you are assigned to one of two different treatment groups to compare the effects of the medications.

  2. Step 2

    <b>treatment phase</b>

    You will receive one of two treatment options based on the group assigned during randomization.

    The first option involves the administration of mifamurtide, which is given through an intravenous use, meaning it is delivered directly into a vein.

    The dose for mifamurtide is 2 mg/m2, which is calculated based on your body surface area.

    The second option involves the administration of sorafenib, which is taken as oral use, meaning it is swallowed as a pill.

    The dose for sorafenib is 800 mg.

  3. Step 3

    <b>monitoring and safety assessment</b>

    Throughout the treatment, physical examinations are performed, including the recording of vital signs (such as blood pressure and heart rate) and laboratory abnormalities (blood tests).

    Safety is monitored by tracking any adverse events, which are side effects or unintended medical occurrences, and serious adverse events, which are more severe medical occurrences.

    The safety follow-up continues from the start of treatment until the end of the designated study period.

  4. Step 4

    <b>evaluation of outcomes</b>

    The effectiveness of the treatment is measured by tracking event-free survival, which is the time from randomization until the first occurrence of death, disease progression, or the return of the disease.

    overall survival is monitored, which tracks the time from randomization to death from any cause.

    progression-free survival is measured, which tracks the time from randomization until the disease gets worse or death occurs.

    The overall response rate is assessed through imaging to see if there is a complete response (disappearance of the tumor) or a partial response (reduction in the size of the tumor).

Who can join the trial?

11 criteria

  • The patient must be between 5 and 30 years old at the time they join the study.
  • The patient must have recovered from any bad side effects caused by previous surgery (medical procedures to remove tissue) or radiotherapy (the use of high-energy rays to kill cancer cells).
  • Patients who have reached puberty and sexual maturity must agree to use effective contraception (methods to prevent pregnancy) during the study and for at least one year after stopping the treatment.
  • The diagnosis of osteosarcoma (a type of bone cancer) must be confirmed by a histopathological examination (a process where a doctor looks at tissue samples under a microscope to identify disease).
  • The patient or their guardian must provide written, informed consent (a signed document showing they understand and agree to take part in the study and the specific treatments).
  • The patient must be classified as high-risk, meaning their condition has a higher chance of worsening or complications.
  • The patient must have a life expectancy (expected remaining lifespan) of at least 12 weeks from the date they sign the consent form.
  • The patient must be considered fit to receive systemic treatment (medication that travels through the entire body via the bloodstream).
  • The patient must be able to swallow tablets.
  • The cancer must be in complete remission (no signs of cancer left) or stable disease (the cancer is not growing or spreading) based on WHO criteria (standardized rules used by doctors to measure cancer status).
  • The patient must have completed major surgery at least 2 weeks before starting the study medication or radiotherapy at least 4 weeks before starting the medication.

Who cannot join the trial?

15 criteria

  • You do not meet all the requirements to join the study.
  • You have had a blood clot, which is a blockage in a blood vessel, in your arteries or veins within the last 6 months. This includes a stroke (when blood flow to the brain is blocked), a transient ischemic attack (a temporary blockage of blood flow to the brain), deep vein thrombosis (a blood clot in a deep vein, usually in the leg), or a pulmonary embolism (a blood clot that travels to the lungs).
  • You have an active or long-term hepatitis B or C infection, which are infections of the liver that may require medicine to treat.
  • You have had any bleeding or hemorrhagic event (excessive bleeding) that is classified as grade 3 or higher, which means the bleeding is severe, such as heavy bleeding that requires medical intervention, within the last 4 weeks.
  • You have been diagnosed with any other type of malignancy, which is another word for cancer, before starting this study.
  • You are currently pregnant, planning to become pregnant, or are breastfeeding.
  • You have any other sudden or ongoing health problems, behaviors, or abnormal laboratory test results (unusual findings in blood or urine tests) that could make the study unsafe or make the results hard to understand.
  • You have previously used the medicine mifamurtide.
  • You have a hypersensitivity, which is an extreme allergic reaction, to the study medicine or any of its parts.
  • You are currently taking other medicines that might react badly with the study drugs.
  • You have lasting toxicity, which refers to harmful side effects, from a previous treatment that makes it unsafe to use the study medicines.
  • You have significant cardiac conduction abnormalities, which are problems with the electrical signals that control your heartbeat, such as long QT syndrome (a specific heart rhythm problem) or a specific measurement of your heart rhythm called QTc that is higher than 480 ms.
  • You have symptoms of congestive heart failure (a condition where the heart cannot pump blood well enough) or a left ventricular ejection fraction (a measurement of how much blood the heart pumps out with each beat) of less than 50%.
  • You need to take high doses of corticosteroids (medicines that reduce inflammation but can affect the immune system) or other immunosuppressive medications (medicines that lower your body's ability to fight infection).
  • You have uncontrolled blood pressure, meaning your blood pressure stays at or above 140/90 mmHg even while taking medicine.
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Investigated drugs

  • Mifamurtide

    is an immunotherapy treatment given through a vein. It is being tested to see if it can help the body's immune system recognize and fight against certain types of cancer cells.

  • Sorafenib

    is a medication taken by mouth. It is used as a standard treatment to help stop the growth of cancer cells by blocking specific signals that the tumor needs to survive and spread.

What is already known about the treatment

  • MIFAMURTIDE

    This medication is an immune-modulating drug administered through an intravenous injection into the bloodstream. It is currently used as an orphan drug, which means it is specifically developed to treat rare medical conditions like high-risk bone cancer. The substance works by stimulating the body's own immune cells to recognize and attack cancer cells more effectively. It belongs to a class of drugs known as immune response modifiers.

  • SORAFENIB

    This medication is taken orally in the form of a pill that is swallowed. It is a well-known treatment used in medical practice to help slow the growth of certain types of cancer. On a molecular level, it works by blocking specific enzymes that help cancer cells grow and form new blood vessels to feed the tumor. It is classified as a multi-kinase inhibitor, which is a type of targeted therapy.

Investigated diseases

  • High-grade osteosarcoma

    This is a type of bone cancer that originates in the cells that form bone tissue. It is characterized by fast-growing cells that often appear very abnormal under a microscope. The disease typically begins in the long bones, such as those in the legs or arms. As it progresses, the cancerous cells can invade nearby soft tissues. The condition may also spread to other parts of the body through the bloodstream.

  • Relapsed/refractory osteosarcoma

    This condition refers to bone cancer that either returns after a period of improvement or does not respond to initial therapy. Relapsed osteosarcoma occurs when the cancer comes back in the same or a different location after being treated. Refractory osteosarcoma describes cancer that continues to grow or remain unchanged despite receiving medical intervention. In both cases, the disease demonstrates persistence or recurrence within the body.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2026-525223-24-00Protocol codeDRAGONFLYEstimated enrolment40 patientsSponsorInstytut Matki I Dziecka

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).