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Cabozantinib treatment for children and young adults with osteosarcoma

A Phase II, Randomized, Open-label Study to Assess the Efficacy, Safety, and Pharmacokinetics (PK) of Maintenance Cabozantinib (XL184) Plus Best Supportive Care (BSC) Versus BSC in Children, Adolescents and Young Adults (AYA) With Unresectable Residual Osteosarcoma Either at Diagnosis or at First Relapse After Standard Treatment

PHASE2 · Ipsen · NCT06341712

This study is testing if adding cabozantinib to regular care can help children and young adults with osteosarcoma that couldn't be fully removed by surgery feel better and manage their cancer.

Quick facts

PhasePHASE2
Study typeInterventional
Enrollment90 (estimated)
Ages5 Years to 30 Years
SexAll
SponsorIpsen (industry)
Drugs / interventionscabozantinib, tivantinib, crizotinib, chemotherapy
Locations74 sites (Los Angeles, California and 73 other locations)
Trial IDNCT06341712 on ClinicalTrials.gov

What this trial studies

This study evaluates the effects of cabozantinib combined with best supportive care versus best supportive care alone in children and young adults with residual osteosarcoma that cannot be completely removed by surgery. Participants must have had a partial response or stable disease after conventional chemotherapy. The study aims to provide insights into the efficacy of cabozantinib in managing this rare type of bone cancer. Best supportive care will be tailored to individual needs, focusing on symptom control and overall well-being.

Who should consider this trial

Good fit: Ideal candidates are children and young adults aged 5 to 30 with high-grade osteosarcoma and residual disease after chemotherapy.

Not a fit: Patients with progressive disease or those who have not received adequate prior chemotherapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve outcomes and quality of life for young patients with osteosarcoma.

How similar studies have performed: Other studies have shown promise with cabozantinib in various cancers, but its specific application in osteosarcoma is still being explored.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Los Angeles, California and 73 other locations

+24 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Osteosarcoma, Osteosarcoma in Children, Osteosarcoma in Adolescents and Young Adults, Cabozantinib, Children, Osteosarcoma in adolescents and young adults, AYA

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.