OsteosarcomaPhase 2CabOSTarNCT06341712
Effects of Maintenance Cabozantinib+BSC Versus BSC in Children and AYA With Osteosarcoma
A Phase 2 study testing Cabozantinib in people with Osteosarcoma and Osteosarcoma in Children, run by Ipsen at 16 sites in 10 countries.
Trial summary
The participants of this study will be children, adolescents, and young adults with residual osteosarcoma, which cannot be removed completely through surgery. Participants will have achieved a partial response or stable disease at the end of conventional chemotherapy. Osteosarcoma is cancer of the bone. Read more about this study
- Who is it for?
- Ages 5 to 30 years
- Conditions the study is for
- Osteosarcoma, Osteosarcoma in Children, Osteosarcoma in Adolescents and Young Adults
- What is being tested?
- Cabozantinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 16 sitesin 10 countries
- Study dates
- 22 Nov 2024to 27 Feb 2026
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
16 sites in 10 countries.
16 sites in 10 countries
Spain4 sites
Hospital de La Santa Creu i Sant Pau
Barcelona
Hospital Universitario Vall d'Hebron
Barcelona
Hospital Infantil Universitario Nino Jesus
Madrid
Hospital Universitari i Politecnic La Fe
Valencia
Canada2 sites
McGill University Health Centre - Centre for Innovative Medicine
Québec
Princess Margaret cancer center
Toronto
Germany2 sites
Universitätsmedizin Mainz
Mainz
Dr. von Haunerschen Kinderspital
München
Italy2 sites
Ospedale Ortopedico Rizzoli di Bologna
Bologna
AOU Città della Salute e della Scienza di Torino
Piemonte
United States1 site
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
About this study
Official title: 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
The participants of this study will be children, adolescents, and young adults with residual osteosarcoma, which cannot be removed completely through surgery.
Participants will have achieved a partial response or stable disease at the end of conventional chemotherapy. Osteosarcoma is cancer of the bone. The cancer cells make immature bone cells, known as osteoid.
Read more
Osteosarcoma is very rare, but it is the most common type of bone cancer in children and teens. It is most common in teens and young adults.
In this study, participants will receive either cabozantinib and best supportive care or the best supportive care alone. Best supportive care will be provided at the investigator's discretion and according to institutional guidelines.
It includes antibiotics, nutritional support, correction of metabolic disorders, optimal symptom control and pain management (including radiotherapy), etc. but does not include tumor specific therapy.
Cabozantinib will be taken by mouth (orally), as a tablet, once a day. Cabozantinib will be provided to participants who tolerate it for as long as their disease does not progress. Participants in the study receiving best supportive care alone may switch to treatment with cabozantinib and best supportive care if their disease progresses and if other eligibility criteria are met.
Participants may withdraw consent to participate at any time.
The estimated duration of the study for participants is 24 months, however a participant could remain in the study longer if demonstrating treatment benefit.
- Being tested
- Cabozantinib, Best Supportive Care (BSC)
- Keywords
- Osteosarcoma, Cabozantinib, Children, Osteosarcoma in adolescents and young adults (AYA), AYA
- Medical subject headings
- Osteosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CabozantinibDrug
Participants will receive cabozantinib orally Once daily (QD) on a continuous dosing schedule for cycles of 28 days.
Best Supportive Care (BSC)Other
Participants will receive BSC. BSC includes antibiotics, nutritional support, correction of metabolic disorders, optimal symptom control and pain management (including palliative radiotherapy), etc. but does not include tumor specific therapy.
Best Supportive Care (BSC)Other
Participants will receive BSC alone. BSC includes antibiotics, nutritional support, correction of metabolic disorders, optimal symptom control and pain management (including palliative radiotherapy), etc. but does not include tumor specific therapy.
| Study group | Role | What they receive |
|---|---|---|
| Arm A: Cabozantinib+ Best supportive care (BSC) | Experimental | Participants will receive cabozantinib and BSC. Receives: Drug: Cabozantinib; Other: Best Supportive Care (BSC) |
| Arm B: Best supportive care (BSC) | Other | Participants will receive BSC alone administered per investigator's discretion and institutional guidelines. Receives: Other: Best Supportive Care (BSC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression-free Survival (PFS) Assessed by Blinded Independent Radiology Committee (BIRC)
PFS was defined as the time from the date of randomization (Cycle 1 Day 1) to the data of first documented disease progression (according to Response Evaluation Criteria in Solid Tumors [RECIST] version 1.1) or the date of death due to any cause, whichever occurred first.
Measured: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Secondary outcomes (17)
Progression-free Survival Rate at 4 Months and 1 Year Assessed by Blinded Independent Radiology Committee
PFS rate at 4 months and 1 year was defined as the percentage of participants who were not progressed by BIRC assessment using RECIST version 1.1 at 4 months and 1 year.
Measured: At 4 months and 1 year after randomization (Cycle 1 Day 1)
Objective Response Rate (ORR) Assessed by Blinded Independent Radiology Committee
ORR was defined as the percentage of participants who had achieved complete response (CR) or partial response (PR) determined by BIRC using RECIST version 1.1. CR was defined as the disappearance of all target lesions, any pathological lymph nodes (whether target or non-target) with reduction in short axis to <10 millimeter (mm). PR was defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.
Measured: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Disease Control Rate (DCR) Assessed by Blinded Independent Radiology Committee
DCR was defined as the percentage of participants who had achieved CR, PR, or stable disease (SD) determined by BIRC using RECIST version 1.1. CR was defined as the disappearance of all target lesions, any pathological lymph nodes (whether target or non-target) with reduction in short axis to <10 mm. PR was defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. SD was defined as neither sufficient shrinkage to qualify for progressive disease (PD). PD was defined as at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this included the baseline sum if that was the smallest on study), in addition to the relative increase of 20%, the sum must have also demonstrated an absolute increase of at least 5 mm.
Measured: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Progression-free Survival Assessed by Investigator
PFS was defined as the time from the date of randomization (Cycle 1 Day 1) to the data of first documented disease progression (according to RECIST version 1.1) or the date of death due to any cause, whichever occurred first.
Measured: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Progression-free Survival Rate at 4 Months and 1 Year Assessed by Investigator
PFS rate at 4 months and 1 year was defined as the percentage of participants who were not progressed by investigator assessment using RECIST version 1.1 at 4 months and 1 year.
Measured: At 4 months and 1 year after randomization (Cycle 1 Day 1)
Objective Response Rate Assessed by Investigator
ORR was defined as the percentage of participants who had achieved CR or PR determined by investigator using RECIST version 1.1. CR was defined as the disappearance of all target lesions, any pathological lymph nodes (whether target or non-target) with reduction in short axis to <10 mm. PR was defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.
Measured: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Disease Control Rate Assessed by Investigator
DCR was defined as the percentage of participants who had achieved CR, PR, or SD determined by investigator using RECIST version 1.1. CR was defined as the disappearance of all target lesions, any pathological lymph nodes (whether target or non-target) with reduction in short axis to <10 mm. PR was defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. SD was defined as neither sufficient shrinkage to qualify for PD. PD was defined as at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this included the baseline sum if that was the smallest on study), in addition to the relative increase of 20%, the sum must have also demonstrated an absolute increase of at least 5 mm.
Measured: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Overall Survival (OS)
The OS was defined as the time from date of randomization to the date of death, from any cause.
Measured: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Overall Survival Rate at 1 Year
The OS rate was defined as the percentage of participants alive at 1 year.
Measured: At 1 year after randomization (Cycle 1 Day 1)
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Treatment-Emergent Adverse Events (TESAEs) and Events to Monitor (ETM)
Adverse events (AEs) were defined as any untoward medical occurrence in a clinical study participant to whom a medicinal product was administered, and which did not necessarily have a causal relationship with the treatment. SAE was defined as any medical occurrence that, at any dose, meets one or more of the criteria listed: resulted in death, was life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; other medically important situations; was a suspected transmission of any infectious agent via an authorized medicinal product. ETM were AEs that may not be serious but were of special importance to a particular drug or class of drugs. TEAEs defined as any AE that started or worsened on or after the first dose of study treatment up to the earliest of death, loss to follow-up, consent withdrawal, last dose of study treatment + 30 days, or data cut-off.
Measured: From the first study treatment administration (Cycle 1 Day 1) up to early termination of the study, approximately 12 months
Area Under the Plasma Concentration Time Curve (AUC) at Steady State of Cabozantinib
Blood samples were planned to be collected at specified timepoints for analysis of AUC.
Measured: Pre-dose and 4 hours post-dose on Cycle 1 Day 1 and Cycle 2 Day 1
Average Concentration (Cavg) at Steady State of Cabozantinib
Blood samples were planned to be collected at specified timepoints for analysis of Cavg.
Measured: Pre-dose and 4 hours post-dose on Cycle 1 Day 1 and Cycle 2 Day 1
Minimum Observed Plasma Drug Concentration (Cmin) at Steady State of Cabozantinib
Blood samples were planned to be collected at specified timepoints for analysis of Cmin.
Measured: Pre-dose and 4 hours post-dose on Cycle 1 Day 1 and Cycle 2 Day 1
Maximal Observed Plasma Drug Concentration (Cmax) at Steady State of Cabozantinib
Blood samples were planned to be collected at specified timepoints for analysis of Cmax.
Measured: Pre-dose and 4 hours post-dose on Cycle 1 Day 1 and Cycle 2 Day 1
Acceptability and Palatability of Cabozantinib Tablets Using a Horizontal Visual Assessment Scale
Acceptability and palatability of cabozantinib tablets in participants were planned to be assessed using a 5-point facial hedonic scale (FHS) with a correlated horizontal 100-point visual assessment scale (VAS). The face scale to assess palatability had 5 response options- very bad, bad, neither good or bad, good, very good. The VAS score ranged from 0 (least palatable) to 100 (most palatable), with higher scores indicating better palatability and acceptability.
Measured: Cycle 1 Day 1
Change From Baseline in Score for All Paediatric Quality of Life (QoL) Inventory (PedsQL) Scales Including Generic Core Scales and Cancer Modules Throughout the Study
The PedsQL a modular instrument was planned to measure health-related quality of life (HRQoL) in participants <18 years of age. The PedsQL Generic Core Scales are multidimensional child self-report and parent proxy-report scales developed as the generic core measure to be integrated with the PedsQL disease specific modules. It assesses pain and hurt; nausea, procedural and treatment anxiety; worry, cognitive problems; perceived physical appearance, and communication. The scores for each subcategories are ranged from 0 (never) to 4 (almost always) .The total scores range from 0 to 100, with higher scores indicating better health related quality of life. Baseline was defined as the last non-missing measurement on or prior to the date of randomization.
Measured: From the first dose of study treatment (Cycle 1 Day 1) up to early termination of the study, approximately 12 months
Change From Baseline in European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) Throughout the Study
EORTC QLQ-C30 was planned to assess HRQoL, functioning, and symptoms in adult participants. It is a 30-item self-administered questionnaire for all cancer types. Questions are grouped into 5 multi-item functional scales (physical, role, emotional, cognitive, and social), 3 multi-item symptom scales (fatigue, pain, and nausea/vomiting), a 2-item global QoL scale, 5 single items assessing additional symptoms commonly reported by cancer patients (dyspnea, loss of appetite, insomnia, constipation, and diarrhea), and 1 item on financial impact of disease. All but 2 questions have 4-point scales ranged from 0 (not at all) and 4 (very much) and 2 questions concerning global health status and QoL have 7-point scales ranged from 0 (very poor) to 7 (excellent). Total scores ranged from 0 to 100, where higher scores indicated better functioning, better HRQoL, or greater level of symptoms. Baseline was defined as last non-missing measurement on or prior to the date of randomization.
Measured: From the first dose of study treatment (Cycle 1 Day 1) up to early termination of the study, approximately 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Apr 2024
- Study start22 Nov 2024
- Primary completion27 Feb 2026
- Study completion27 Feb 2026
Why it stopped: The sponsor has decided to terminate the CabOSTar study due to ongoing recruitment challenges
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for OsteosarcomaStudies of the same condition, open and under way.
- More trials run by IpsenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06341712
- Sponsor's protocol ID
- CLIN-60000-461
- Other IDs
- 2023-506229-12-00, 2023
- Acronym
- CabOSTar
- Status last verified
- Oct 2026
- First submitted
- 4 Mar 2024
- Last updated
- 29 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06341712
- Acronym / sponsor ID
- CabOSTar / CLIN-60000-461
- Keywords
- Osteosarcoma, Cabozantinib, Children, Osteosarcoma in adolescents and young adults (AYA), AYA
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The sponsor has decided to terminate the CabOSTar study due to ongoing recruitment challenges
- Enrolment
- 10 participants (actual)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- 22 Nov 2024 (actual)
- Primary completion
- 27 Feb 2026 (actual)
- Study completion
- 27 Feb 2026 (actual)
- First posted
- 2 Apr 2024 (actual)
- Results
- Posted 29 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The participants of this study will be children, adolescents, and young adults with residual osteosarcoma, which cannot be removed completely through surgery.
Participants will have achieved a partial response or stable disease at the end of conventional chemotherapy. Osteosarcoma is cancer of the bone. The cancer cells make immature bone cells, known as osteoid.
Osteosarcoma is very rare, but it is the most common type of bone cancer in children and teens. It is most common in teens and young adults.
In this study, participants will receive either cabozantinib and best supportive care or the best supportive care alone. Best supportive care will be provided at the investigator's discretion and according to institutional guidelines.
It includes antibiotics, nutritional support, correction of metabolic disorders, optimal symptom control and pain management (including radiotherapy), etc. but does not include tumor specific therapy.
Cabozantinib will be taken by mouth (orally), as a tablet, once a day. Cabozantinib will be provided to participants who tolerate it for as long as their disease does not progress. Participants in the study receiving best supportive care alone may switch to treatment with cabozantinib and best supportive care if their disease progresses and if other eligibility criteria are met.
Participants may withdraw consent to participate at any time.
The estimated duration of the study for participants is 24 months, however a participant could remain in the study longer if demonstrating treatment benefit.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A: Cabozantinib+ Best supportive care (BSC) | Experimental | Drug: Cabozantinib, Other: Best Supportive Care (BSC)Participants will receive cabozantinib and BSC. |
| Arm B: Best supportive care (BSC) | Other | Other: Best Supportive Care (BSC)Participants will receive BSC alone administered per investigator's discretion and institutional guidelines. |
Who may qualify
- Ages
- 5 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (14 listed)
- 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.
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CabozantinibParticipants will receive cabozantinib orally Once daily (QD) on a continuous dosing schedule for cycles of 28 days. | Drug | Arm A: Cabozantinib+ Best supportive care (BSC) |
| Best Supportive Care (BSC)Participants will receive BSC. BSC includes antibiotics, nutritional support, correction of metabolic disorders, optimal symptom control and pain management (including palliative radiotherapy), etc. but does not include tumor specific therapy. | Other | Arm A: Cabozantinib+ Best supportive care (BSC) |
| Best Supportive Care (BSC)Participants will receive BSC alone. BSC includes antibiotics, nutritional support, correction of metabolic disorders, optimal symptom control and pain management (including palliative radiotherapy), etc. but does not include tumor specific therapy. | Other | Arm B: Best supportive care (BSC) |
Primary outcome
Progression-free Survival (PFS) Assessed by Blinded Independent Radiology Committee (BIRC)
Time frame: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
PFS was defined as the time from the date of randomization (Cycle 1 Day 1) to the data of first documented disease progression (according to Response Evaluation Criteria in Solid Tumors [RECIST] version 1.1) or the date of death due to any cause, whichever occurred first.
17 secondary outcomes
Progression-free Survival Rate at 4 Months and 1 Year Assessed by Blinded Independent Radiology Committee
Time frame: At 4 months and 1 year after randomization (Cycle 1 Day 1)
Objective Response Rate (ORR) Assessed by Blinded Independent Radiology Committee
Time frame: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Disease Control Rate (DCR) Assessed by Blinded Independent Radiology Committee
Time frame: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Progression-free Survival Assessed by Investigator
Time frame: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Progression-free Survival Rate at 4 Months and 1 Year Assessed by Investigator
Time frame: At 4 months and 1 year after randomization (Cycle 1 Day 1)
Objective Response Rate Assessed by Investigator
Time frame: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Disease Control Rate Assessed by Investigator
Time frame: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Overall Survival (OS)
Time frame: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months
Overall Survival Rate at 1 Year
Time frame: At 1 year after randomization (Cycle 1 Day 1)
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Treatment-Emergent Adverse Events (TESAEs) and Events to Monitor (ETM)
Time frame: From the first study treatment administration (Cycle 1 Day 1) up to early termination of the study, approximately 12 months
Area Under the Plasma Concentration Time Curve (AUC) at Steady State of Cabozantinib
Time frame: Pre-dose and 4 hours post-dose on Cycle 1 Day 1 and Cycle 2 Day 1
Average Concentration (Cavg) at Steady State of Cabozantinib
Time frame: Pre-dose and 4 hours post-dose on Cycle 1 Day 1 and Cycle 2 Day 1
Minimum Observed Plasma Drug Concentration (Cmin) at Steady State of Cabozantinib
Time frame: Pre-dose and 4 hours post-dose on Cycle 1 Day 1 and Cycle 2 Day 1
Maximal Observed Plasma Drug Concentration (Cmax) at Steady State of Cabozantinib
Time frame: Pre-dose and 4 hours post-dose on Cycle 1 Day 1 and Cycle 2 Day 1
Acceptability and Palatability of Cabozantinib Tablets Using a Horizontal Visual Assessment Scale
Time frame: Cycle 1 Day 1
Change From Baseline in Score for All Paediatric Quality of Life (QoL) Inventory (PedsQL) Scales Including Generic Core Scales and Cancer Modules Throughout the Study
Time frame: From the first dose of study treatment (Cycle 1 Day 1) up to early termination of the study, approximately 12 months
Change From Baseline in European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) Throughout the Study
Time frame: From the first dose of study treatment (Cycle 1 Day 1) up to early termination of the study, approximately 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ipsen Medical Director study director
Ipsen
16 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cabozantinib in people with Osteosarcoma and Osteosarcoma in Children, run by Ipsen at 16 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in Spain, Canada, Germany, Italy, United States and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progression-free Survival (PFS) Assessed by Blinded Independent Radiology Committee (BIRC) (time frame: Baseline (Cycle 1 Day 1) and every 8 weeks during the treatment period and then every 3 months during the follow up period until disease progression or death from any cause, whichever occurs first. The maximum study duration was approximately 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Oct 2026.