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Study on the Effectiveness and Safety of Regorafenib for Patients with Resistant Primary Bone Tumors

Verified siteRegistered drugNo placebo
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What is this trial about?

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

This clinical trial is focused on studying the effects of a medication called Regorafenib on patients with a type of cancer known as refractory primary bone tumors. These tumors are a form of cancer that originates in the bones and have not responded to previous treatments. The purpose of the study is to evaluate how effective and safe Regorafenib is for these patients.

Participants in the study will take Regorafenib in the form of a film-coated tablet, which is taken orally. The study will monitor patients over a period to see how the medication affects their condition. This includes observing any changes in the tumor, as well as any side effects that may occur. The study aims to determine if Regorafenib can help control the disease and improve the quality of life for patients.

Throughout the study, various health checks will be conducted, such as laboratory tests and heart monitoring, to ensure the safety of the participants. The study will also look at how the body processes the medication, including how long it takes to reach a steady level in the bloodstream. This information will help researchers understand the best way to use Regorafenib in treating refractory primary bone tumors.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on criteria such as age, diagnosis of Ewing's sarcoma or osteosarcoma, and previous treatment failure.

    Written informed consent is required before starting any procedures related to the study.

  2. Step 2

    Treatment initiation

    The treatment involves taking regorafenib, which is administered orally.

    The dosage is determined to achieve drug exposure similar to that recommended for adults, especially for patients aged 9 to 18 years.

  3. Step 3

    Treatment duration

    The treatment with regorafenib continues as per the study protocol, with regular monitoring of health and response to the medication.

    Effective contraception is required throughout the treatment period and for at least 2 years after discontinuation for patients in puberty.

  4. Step 4

    Monitoring and assessments

    Regular assessments include monitoring vital signs, laboratory tests, echocardiography, and ECG to evaluate the safety of regorafenib.

    Adverse events are recorded and analyzed to ensure patient safety.

  5. Step 5

    End of study

    The study is estimated to conclude by December 31, 2025.

    Final assessments will determine the efficacy and safety of regorafenib in treating refractory primary bone tumors.

Who can join the trial?

7 criteria

  • Age must be more than 9 years and up to 21 years at the time of joining the study.
  • Must have a type of bone cancer called Ewing's sarcoma or osteosarcoma, confirmed by a test that looks at tissue under a microscope.
  • Must have experienced treatment failure, which means the cancer did not respond to previous treatments or came back, identified no earlier than 30 days before starting the study treatment.
  • Must provide written, informed consent to participate in the study before any study procedures begin, including treatment with the study drug, regorafenib.
  • Must have a life expectancy of at least 12 weeks from the time of signing the informed consent.
  • Must be able to swallow a tablet.
  • If in puberty, must agree to use effective birth control during the treatment period with regorafenib and for at least 2 years after stopping the treatment.

Who cannot join the trial?

7 criteria

  • Patients who have not been diagnosed with refractory primary bone tumors. This means the study is only for those whose bone tumors do not respond to treatment.
  • Patients who are not within the specified age range for the study. The study is only for certain age groups.
  • Patients who are not able to follow the study procedures or take the study medication as required.
  • Patients who have other medical conditions that might interfere with the study or make it unsafe for them to participate.
  • Patients who are pregnant or breastfeeding, as the study may not be safe for them or their babies.
  • Patients who are participating in another clinical trial at the same time, as this could affect the results of the study.
  • Patients who have allergies or reactions to the study medication or similar medications.
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Investigated drugs

Regorafenib is a medication being studied for its effectiveness and safety in treating patients with primary bone tumors that have not responded to other treatments. It works by blocking certain proteins that help cancer cells grow and spread, potentially slowing down or stopping the progression of the tumor.

What is already known about the treatment

Regorafenib – This medication is taken orally in the form of tablets. It is currently being studied in clinical trials for its effectiveness and safety in treating refractory primary bone tumors. Regorafenib is primarily used for its anti-cancer properties, particularly in cases where other treatments have failed. It works by inhibiting multiple protein kinases, which are involved in tumor growth and the development of blood vessels that supply the tumor. Regorafenib is classified as a multi-kinase inhibitor in pharmacology.

Investigated diseases

Refractory Primary Bone Tumors – These are bone tumors that do not respond to standard treatments. They originate in the bone and are considered primary because they start there rather than spreading from another part of the body. The term "refractory" indicates that the tumors persist despite treatment efforts. These tumors can cause pain, swelling, and may weaken the bone, leading to fractures. As they progress, they may grow larger and potentially spread to other areas. The progression can vary depending on the specific type of bone tumor and individual patient factors.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-514720-17-00Protocol codeREGBONEEstimated enrolment30 patientsSponsorInstytut Matki I Dziecka

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