A Phase 2 interventional study of Cabozantinib and Best Supportive Care (BSC) in Osteosarcoma, Osteosarcoma in Children and Osteosarcoma in Adolescents and Young Adults, sponsored by Ipsen. Terminated at 16 sites in 10 countries. Open to participants aged 5 Years to 30 Years. Per ClinicalTrials.gov, last updated 2026-03-31.
Sponsored by Ipsen · Phase 2, Interventional, and 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.
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.
437 studies on the registry are indexed under Osteosarcoma; 116 are open to participants now.
This study's enrollment of 10 is below the median of 42 across 325 interventional studies indexed under Osteosarcoma.
Browse Osteosarcoma studies →Ipsen is the lead sponsor of 282 studies on the registry; 16 are open to participants now.
Of its 24 completed or terminated interventional studies of FDA-regulated products, 19 (79%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria :
Participants will receive cabozantinib and BSC.
Drug: Cabozantinib · Other: Best Supportive Care (BSC)
Participants will receive BSC alone administered per investigator's discretion and institutional guidelines.
Other: Best Supportive Care (BSC)
Participants will receive cabozantinib orally Once daily (QD) on a continuous dosing schedule for cycles of 28 days.
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.
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.
Progression-free Survival (PFS) assessed by Blinded Independent Radiology Committee (BIRC)
PFS defined as the time from the date of randomization to the date of first documented disease progression or the date of death due to any cause, whichever occurs first.
Time frame: From randomization until disease progression or death from any cause, whichever occurs first (approximately 34 months).
Progression-free survival (PFS) rate assessed by BIRC
PFS rate at 4 months and 1 year was defined as the probability that participants have not progressed by BIRC assessment and remain alive at 4 months and 1 year.
Time frame: 4 months and 1 year after randomization.
Objective response rate (ORR) assessed by BIRC
ORR defined as the proportion of participants who have achieved complete response (CR) or partial response (PR) determined by BIRC.
Time frame: Approximately 34 months after randomization.
Disease control rate (DCR) assessed by BIRC
Defined as the proportion of participants who have achieved CR, PR, or stable disease (SD) determined by BIRC
Time frame: Approximately 34 months after randomization.
PFS assessed by investigator
Defined as the time from the date of randomization to the date of first documented disease progression determined by investigator or the date of death due to any cause, whichever occurs first
Time frame: From randomization until disease progression or death from any cause, whichever occurs first (approximately 34 months).
PFS rate assessed by investigator
Defined as the probability that participants have not progressed by investigator assessment and remain alive at 4 months and 1 year.
Time frame: At 4 months and 1 year after randomization.
ORR assessed by investigator
Defined as the proportion of participants who have achieved complete response (CR) or partial response (PR) determined by investigator using RECIST version 1.1.
Time frame: Approximately 34 months after randomization.
DCR assessed by investigator
Defined as the proportion of participants who have achieved CR, PR, or SD determined by investigator.
Time frame: Approximately 34 months after randomization.
Overall survival (OS)
Defined as the time from date of randomization to the date of death, from any cause
Time frame: From randomization until death or last contact (approximately 34 months).
1-year overall survival rate
Defined as the probability participants alive at 1 year.
Time frame: At 1 year after randomization.
Percentage of participants with Treatment Emergent Adverse Event (TEAEs) and Adverse Events of Special Interest (AESIs).
An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. AESIs are AEs that may not be serious but are of special importance to a particular drug or class of drugs.
Time frame: From screening to 30 days after last dose.
Area Under Curve (AUC) at steady state.
Time frame: At Day 1 of week 1 and Day 1 of week 5.
Average concentration (Cavg) at steady state
Time frame: At Day 1 of week 1 and Day 1 of week 5.
Minimum concentration (Cmin) at steady state
Time frame: At Day 1 of week 1 and Day 1 of week 5.
Maximal concentration (Cmax) at steady state
Time frame: At Day 1 of week 1 and Day 1 of week 5.
Acceptability and palatability in children and adolescents assessed using a horizontal visual assessment scale.
Five-point Facial Hedonic Scale (FHS) with a correlated 100-point horizontal Visual Analog Scale (VAS) (FHS/VAS-5) will be used to assess acceptability and palatability in children and adolescents. Final scores range from 0 to 100, with higher scores indicating better palatability and acceptability.
Time frame: Day of first dose.
Change from baseline in score for all Paediatric QoL Inventory (PedsQL) Scales including Generic Core Scales and Cancer Modules.
The PedsQL is a modular instrument designed to measure health-related quality of life in children and adolescent. 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. The PedsQL cancer modules was designed to measure paediatric cancer specific HRQoL. Final total scores range from 0 to 100, with higher scores indicating better health related quality of life.
Time frame: From screening to 30 days after last dose.
Change from baseline in European Organization for the Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) (EORTC QLQ-C30) for adult participants
The EORTC QLQ-C30 was developed by the EORTC Quality of Life Group to assess HRQoL, functioning, and symptoms in cancer clinical trials. It is a 30-item self-administered questionnaire for all cancer types. Final scores range from 0 to 100, with higher scores indicating better health related quality of life. A high score for a functional scale represents a high / healthy level of functioning, a high score for the global health status / QoL represents a high QoL, but a high score for a symptom scale / item represents a high level of symptomatology / problems.
Time frame: From screening to 30 days after last dose.
Plan to share: 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.
No publications or documents are linked to this record.
This study is terminated, as verified in Mar 2026. You cannot join it, but the record below documents what was studied.
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