Effects of Maintenance Cabozantinib+BSC Versus BSC in Children and AYA With Osteosarcoma
- Conditions
- OsteosarcomaOsteosarcoma in ChildrenOsteosarcoma in Adolescents and Young Adults
- Interventions
- Other: Best Supportive Care (BSC)
- Registration Number
- NCT06341712
- Lead Sponsor
- Ipsen
- Brief 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. 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 90
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Arm A: Cabozantinib+ Best supportive care (BSC) Best Supportive Care (BSC) Participants will receive cabozantinib and BSC. Arm B: Best supportive care (BSC) Best Supportive Care (BSC) Participants will receive BSC alone administered per investigator's discretion and institutional guidelines. Arm A: Cabozantinib+ Best supportive care (BSC) Cabozantinib Participants will receive cabozantinib and BSC.
- Primary Outcome Measures
Name Time Method Progression-free Survival (PFS) assessed by Blinded Independent Radiology Committee (BIRC) From randomization until disease progression or death from any cause, whichever occurs first (approximately 34 months). 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.
- Secondary Outcome Measures
Name Time Method Objective response rate (ORR) assessed by BIRC Approximately 34 months after randomization. ORR defined as the proportion of participants who have achieved complete response (CR) or partial response (PR) determined by BIRC.
Percentage of participants with Treatment Emergent Adverse Event (TEAEs) and Adverse Events of Special Interest (AESIs). From screening to 30 days after last dose. 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.
Disease control rate (DCR) assessed by BIRC Approximately 34 months after randomization. Defined as the proportion of participants who have achieved CR, PR, or stable disease (SD) determined by BIRC
PFS rate assessed by investigator At 4 months and 1 year after randomization. Defined as the probability that participants have not progressed by investigator assessment and remain alive at 4 months and 1 year.
DCR assessed by investigator Approximately 34 months after randomization. Defined as the proportion of participants who have achieved CR, PR, or SD determined by investigator.
Area Under Curve (AUC) at steady state. At Day 1 of week 1 and Day 1 of week 5. Progression-free survival (PFS) rate assessed by BIRC 4 months and 1 year after randomization. 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.
ORR assessed by investigator Approximately 34 months after randomization. Defined as the proportion of participants who have achieved complete response (CR) or partial response (PR) determined by investigator using RECIST version 1.1.
Average concentration (Cavg) at steady state At Day 1 of week 1 and Day 1 of week 5. 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 From screening to 30 days after last dose. 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.
PFS assessed by investigator From randomization until disease progression or death from any cause, whichever occurs first (approximately 34 months). 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
Overall survival (OS) From randomization until death or last contact (approximately 34 months). Defined as the time from date of randomization to the date of death, from any cause
1-year overall survival rate At 1 year after randomization. Defined as the probability participants alive at 1 year.
Minimum concentration (Cmin) at steady state At Day 1 of week 1 and Day 1 of week 5. Maximal concentration (Cmax) at steady state At Day 1 of week 1 and Day 1 of week 5. Change from baseline in score for all Paediatric QoL Inventory (PedsQL) Scales including Generic Core Scales and Cancer Modules. From screening to 30 days after last dose. 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.
Acceptability and palatability in children and adolescents assessed using a horizontal visual assessment scale. Day of first dose. 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.
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Trial Locations
- Locations (74)
University of Southern California (USC) - Norris Cancer Hospital
🇺🇸Los Angeles, California, United States
Stanford University and Lucile Packard Children's Hospital
🇺🇸Palo Alto, California, United States
University of Florida Health Shands Children's Hospital
🇺🇸Gainesville, Florida, United States
Levine Children's Hospital (LCH)
🇺🇸Charlotte, North Carolina, United States
Cincinnati Children's Hospital Medical Center - Cancer and Blood Diseases Institute (CBDI)
🇺🇸Cincinnati, Ohio, United States
MD Anderson Main Campus
🇺🇸Houston, Texas, United States
Children's Hospital of the King's Daughters
🇺🇸Norfolk, Virginia, United States
St. Anna Kinderspital Zentrum fuer Kinder- und Jugendheilkunde
🇦🇹Vienna, Austria
Cliniques universitaires Saint-Luc
🇧🇪Bruxelles, Belgium
University Hospital Gent
🇧🇪Ghent, Belgium
Scroll for more (64 remaining)University of Southern California (USC) - Norris Cancer Hospital🇺🇸Los Angeles, California, United States