Cancer drug cabozantinib tested in kids and young adults with bone cancer
NCT ID NCT06341712
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested the drug cabozantinib in children, teens, and young adults with osteosarcoma (a bone cancer) that could not be fully removed by surgery. Participants who had stable or partially improved disease after chemotherapy received either cabozantinib plus best supportive care or best supportive care alone. The goal was to see if cabozantinib could delay cancer progression. The trial was terminated early and enrolled only 10 people, so its findings are very limited.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- cabozantinib (a cancer drug taken as a tablet)
- What this could lead to
- If it works, this could offer a way to delay cancer growth in young people with osteosarcoma that cannot be surgically removed.
- What could go wrong
- This trial was terminated early with only 10 participants, so results are very limited. Cabozantinib may cause side effects like fatigue, diarrhea, or high blood pressure, and it is not a cure.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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10 people
The number who actually took part.
- Started
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Nov 2024
- Finished
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Feb 2026
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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5 to 30 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria : * Participants must be ≥5 and ≤30 years of age at the time of study entry. * Histologically or cytologically confirmed diagnosis of high-grade osteosarcoma as defined by a local pathologist * Participants with unresectable residual disease after standard chemotherapy treatment at diagnosis or first relapse (treated with systemic chemotherapy). A minimum of 4 cycles of systemic chemotherapy (or minimum of 2 cycles if chemotherapy was stopped early due to toxicity) must have been received. * Measurable residual or evaluable disease by RECIST version 1.1. Participants will be considered with evaluable disease if they have only non-measurable disease as per RECIST version 1.1 criteria. * Absence of Progressive Disease (PD) (defined by the investigator according to RECIST version 1.1) at study entry. Note, the two most recent radiological evaluations (e.g. computerised tomography (CT) or magnetic resonance resonance imaging (MRI) scan) including the one following completion of chemotherapy should be available later to facilitate BIRC review. * Chemotherapy must be the last anticancer treatment received by participants before study entry and must have been completed at least 4 weeks but no longer than 2 months before randomization. * Participants must have recovered to Grade ≤1, except for alopecia, ototoxicity, and Grade ≤2 peripheral neuropathy, per Common Terminology Criteria for Adverse Events (CTCAE) version 5.0) from the acute toxic effects of all prior anticancer therapy at study entry, unless AEs are clinically non significant and/or stable on supportive therapy, per investigator clinical judgment. * Life expectancy \>6 months. * Performance level: participants must have a Lansky or Karnofsky performance status score of ≥70 corresponding to Eastern Cooperative Oncology Group (ECOG) categories 0-1. * Adequate organ and marrow function. * Adequately controlled blood pressure (BP) with or without antihypertensive medications. * Male and/or female (according to their reproductive organs and functions assigned by chromosomal complement) (FDA 2016) * Contraception and barriers as well as pregnancy testing is required as appropriate for the age and sexual activity of pediatric participants and as required by local regulations. * All participants (typically ≥18 years) and/or their parents or legal guardians must sign a written informed consent and assent must be obtained from minor participants according to local guidelines. Exclusion Criteria : * Low grade osteosarcoma and periosteal osteosarcoma * Previous treatment with cabozantinib or another Mesenchymal-epithelial transition (MET)/hepatocyte growth factor (HGF) inhibitor (e.g., tivantinib, crizotinib). * Receipt of any type of small molecule kinase inhibitor (including investigational kinase inhibitor) within 2 weeks or 5 half-lives of the agent, whichever is longer, before first dose of study intervention. * Receipt of any type of cytotoxic, biologic or other systemic anticancer therapy (including investigational) within 4 weeks before first dose of study intervention (or washout of at least 5 half-lives, whichever is shorter). * Known brain metastases or cranial epidural disease unless adequately treated with radiotherapy and/or surgery (including radiosurgery or major surgery e.g., removal or biopsy of brain metastasis) and stable for at least 4 weeks prior to randomization. Eligible participants must be neurologically asymptomatic and without systemic corticosteroid treatment at the time of randomization. Note: Participants with a known seizure disorder who are receiving non-enzyme inducing anticonvulsants and have well-controlled seizures on a stable dose of anti-convulsant may be enrolled. * Participants who have an uncontrolled/active infection requiring systemic therapy. * Participants who are unable to swallow intact tablets. * Participants with uncontrolled, significant intercurrent or recent illness. * Previously identified allergy or hypersensitivity to components of the study treatment formulations. * Any other active malignancy at time of first dose of study intervention or diagnosis of another malignancy within 3 years prior to first dose of study intervention that requires active treatment. * Pregnancy or breast-feeding. * Participants who in the opinion of the investigator may not be able to comply with the requirements of the study are not eligible * Major surgery (eg, orthopaedic surgery, removal or biopsy of brain metastasis) within 8 weeks before randomization. Complete wound healing from major surgery must have occurred 4 weeks before randomization and from minor surgery (eg, simple excision, tooth extraction) at least 10 days before randomization. Participants with clinically relevant ongoing complications from prior surgery are not eligible.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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AOU Città della Salute e della Scienza di Torino
Piemonte, Italy
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Amsterdam UMC - Locatie AMC
Amsterdam, Netherlands
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Birmingham Children's Hospital
Birmingham, United Kingdom
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Centre Oscar Lambret
Lille, France
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Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507, United States
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Dr. von Haunerschen Kinderspital
München, Germany
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Hospital Infantil Universitario Nino Jesus
Madrid, Spain
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Hospital Universitari i Politecnic La Fe
Valencia, Spain
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Hospital Universitario Vall d'Hebron
Barcelona, Spain
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Hospital de La Santa Creu i Sant Pau
Barcelona, Spain
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Instytut Matki i Dziecka
Warsaw, Poland
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McGill University Health Centre - Centre for Innovative Medicine
Québec, Canada
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Ospedale Ortopedico Rizzoli di Bologna
Bologna, Italy
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Princess Margaret cancer center
Toronto, Canada
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University Hospital Gent
Ghent, Belgium
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Universitätsmedizin Mainz
Mainz, Germany
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