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Efficacy and Safety of Maintenance Cabozantinib Plus Best Supportive Care in Pediatric and AYA Patients with Unresectable Residual Osteosarcoma

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 osteosarcoma, a type of bone cancer, in children, adolescents, and young adults. The study will use a medication called cabozantinib, which is also known by its code name XL-184. Cabozantinib is taken in the form of film-coated tablets. The purpose of the study is to evaluate how effective cabozantinib is when used as a maintenance treatment, alongside the best supportive care, compared to just the best supportive care alone. This is aimed at individuals with osteosarcoma that cannot be surgically removed, either at the time of diagnosis or after the first relapse following standard treatment.

Participants in the study will be randomly assigned to receive either cabozantinib plus supportive care or just supportive care. The study will monitor the participants over a period to see how the treatment affects the progression of the disease. The trial will also assess the safety of cabozantinib and how the body processes the drug, which is known as pharmacokinetics. The study will involve regular check-ups and assessments to track the health and progress of the participants.

The trial aims to provide valuable information on whether cabozantinib can help improve the outcomes for young people with osteosarcoma. The study will last for several months, and participants will be closely monitored throughout the process to ensure their safety and to gather data on the effectiveness of the treatment. The results of this study could potentially lead to better treatment options for those affected by this challenging condition.

The research process

The trial runs in 9 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization

    After you have joined the study, you are assigned a random group. the randomization decides whether you will receive cabozantinib plus best supportive care (bsc) or bsc alone.

  2. Step 2

    Assignment to treatment arm

    If you are placed in the cabozantinib arm, you will be given the medication to take at home.

    If you are placed in the bsc only arm, you will continue to receive supportive care without the study drug.

  3. Step 3

    Start of medication

    For participants receiving cabozantinib, the prescribed dose is one 60 mg film‑coated tablet taken by mouth once each day.

    The tablet is swallowed with water and can be taken with or without food.

    You will continue taking the tablet every day until disease progression, death, or a decision is made to stop because of side effects.

  4. Step 4

    First‑dose assessment

    On the day you take the first tablet, study staff will ask you to rate how acceptable and how pleasant the medicine tastes using a five‑point facial scale and a visual analog scale.

    This assessment helps determine whether the medicine is suitable for children and adolescents.

  5. Step 5

    Pharmacokinetic sampling

    Blood samples are collected on cycle 1 day 1 (the first day you take the tablet) and again on cycle 2 day 1 (approximately 28 days later).

    These samples measure how much of the drug is in your blood at steady state, including the minimum, average, and maximum concentrations.

  6. Step 6

    Regular safety and efficacy visits

    You will attend clinic visits at scheduled intervals. during each visit, health professionals will check for side effects, perform physical examinations, and may draw blood for laboratory tests.

    Imaging studies (such as scans) are performed to evaluate disease status. the first major assessment occurs at 4 months after randomization, followed by another at 1 year, and then periodically until the study ends (approximately 34 months).

  7. Step 7

    Monitoring for adverse events

    Any new symptom or health problem that appears from the time of screening until 30 days after the last dose is recorded as a treatment‑emergent adverse event.

    Serious adverse events are followed until they are resolved or stable, even after the medication is stopped.

  8. Step 8

    Continuation of treatment

    You will keep taking the daily tablet as long as the disease does not progress, you do not experience unacceptable toxicity, and you remain alive.

    The expected maximum duration of treatment is about 34 months, but it may be shorter if progression or other stopping criteria occur.

  9. Step 9

    Final follow‑up

    After stopping the medication, you will continue to be followed for overall survival. the study records whether participants are alive at 1 year and up to the final study cut‑off (approximately 34 months after randomization).

Who can join the trial?

14 criteria

  • Be between 5 and 30 years old when you join the study.
  • Have adequate organ and bone‑marrow function – meaning your heart, liver, kidneys, and blood‑forming tissue are working well enough for the trial.
  • Have controlled blood pressure, whether you take medicines for it or not.
  • Both males and females are eligible, based on their chromosomal sex.
  • Use appropriate birth control methods and have a pregnancy test if needed for your age and sexual activity.
  • Provide a written informed consent (and a signed assent if you are a minor) agreeing to take part in the study.
  • Have a diagnosis of high‑grade osteosarcoma confirmed by a pathologist who examined your tissue under a microscope.
  • Have disease that cannot be removed by surgery after standard chemotherapy or after a first relapse, and have received at least 4 cycles of chemotherapy (or at least 2 cycles if treatment stopped early because of side effects).
  • Have tumor that can be measured or evaluated on scans according to RECIST version 1.1 (a set of rules doctors use to track tumor size).
  • Not have disease progression (PD) at the start of the study; the two most recent scans (either CT or MRI) must be available for review.
  • The last chemotherapy must have been finished at least 4 weeks but no more than 2 months before you are randomly assigned to a treatment group.
  • Have recovered from side effects of previous treatment to a severity of Grade 1 or less (except hair loss, ear toxicity, and nerve problems, which can be up to Grade 2); grades are a way doctors rate how strong side effects are.
  • Have a life expectancy longer than 6 months, meaning doctors expect you to live at least that long.
  • Have a performance score of 70 or higher on the Lansky or Karnofsky scale (which corresponds to ECOG 0‑1), indicating you can carry out most normal daily activities.

Who cannot join the trial?

14 criteria

  • Having low‑grade osteosarcoma (a slower‑growing tumor) or periosteal osteosarcoma (a tumor that starts on the outer surface of the bone) means you cannot join the study.
  • Having any other active cancer now, or having been diagnosed with another cancer that needed treatment within the past three years, makes you ineligible.
  • Being pregnant or breastfeeding prevents participation.
  • If the doctor thinks you may not be able to follow the study rules, you cannot take part.
  • Having major surgery (such as major bone surgery or removal/biopsy of a brain tumor) within eight weeks before randomization, or not having full wound healing (four weeks after major surgery or ten days after minor surgery), disqualifies you.
  • Having ongoing problems from a previous surgery that are medically important also excludes you.
  • If you have already been treated with cabozantinib or any other drug that blocks the MET/hepatocyte growth factor (HGF) pathway (examples: tivantinib, crizotinib), you cannot join.
  • Taking any small‑molecule kinase inhibitor (a type of targeted cancer drug, even experimental ones) within two weeks—or within five drug “half‑lives,” whichever is longer—before the first study dose excludes you.
  • Receiving any chemotherapy, biologic therapy, or other systemic cancer treatment (including experimental drugs) within four weeks before the first study dose—or not having a washout of at least five half‑lives, whichever is shorter—means you are not eligible.
  • Having cancer that has spread to the brain (brain metastases) or to the space just outside the brain (cranial epidural disease), unless it has been treated and stable for at least four weeks, prevents participation.
  • Having an uncontrolled or active infection that needs medication excludes you.
  • Being unable to swallow whole tablets makes you ineligible.
  • Having a serious, uncontrolled, or recent illness that could interfere with the study also disqualifies you.
  • Having a known allergy or severe reaction to any part of the study medication prevents participation.
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Investigated drugs

  • Cabozantinib

    is an oral medicine that blocks certain proteins that help cancer cells grow and spread. In this study it is given after the standard treatment for osteosarcoma to try to keep the disease from getting worse and to lengthen the time before the cancer returns.

  • Best supportive care (BSC)

    includes all the non‑cancer‑specific treatments that help patients feel better and stay as healthy as possible. This can involve pain relief, blood transfusions, nutrition help, infection prevention, and other measures that support daily living while the disease is being monitored.

What is already known about the treatment

  • CABOMETYX 20 mg film-coated tablets

    This medication is taken by mouth as a film‑coated tablet. It is an approved cancer drug that is widely studied in medical literature. It is used mainly for kidney and thyroid cancers and is now being tested as a maintenance treatment for osteosarcoma in children and young adults. It works by blocking several proteins called tyrosine kinases (such as MET, VEGFR and AXL), which slows tumor growth and the formation of new blood vessels; it belongs to the class of tyrosine‑kinase inhibitor drugs.

  • CABOMETYX 60 mg film-coated tablets

    This medication is taken by mouth as a film‑coated tablet. It is an approved cancer drug with extensive research and clinical use. It is indicated for kidney and thyroid cancers and is currently being evaluated as a maintenance therapy for osteosarcoma in children, adolescents and young adults. It blocks multiple tyrosine‑kinase enzymes (including MET, VEGFR and AXL), reducing tumor spread and blood‑vessel growth, and is classified as a tyrosine‑kinase inhibitor.

Investigated diseases

  • Osteosarcoma in Children

    Osteosarcoma is a cancer that starts in the bone, most often in the long bones around the knee. In children it usually appears during rapid growth periods. The tumor can enlarge, causing pain and swelling, and may break through the bone surface. Over time it can extend into nearby soft tissue. If it spreads, it can reach other bones or the lungs.

  • Osteosarcoma in Adolescents and Young Adults

    This form also begins in the bone, commonly affecting the thigh bone or shin. It typically emerges during the teenage years when growth plates are closing. The tumor grows, leading to persistent pain and sometimes a lump that can be felt under the skin. It may expand beyond the bone into surrounding muscles. In some cases it can travel through the bloodstream to distant sites.

  • Osteosarcoma

    Osteosarcoma is a malignant bone tumor that most often develops in the long bones of the arms or legs. It starts when abnormal bone cells multiply uncontrollably. The mass can cause pain, swelling, and reduced movement of the affected limb. As it grows, it can break through the outer bone layer and invade nearby tissues. It may also spread to other parts of the body through the blood.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-506229-12-00Protocol codeCLIN-60000-461Estimated enrolment106 patientsSponsorIpsen Innovation

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