CabOSTar

• Research type

  Research Study

• Full 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.

• IRAS ID

  1009468

• Contact name

  Sophie Clavier

• Contact email

  sophie.clavier@ipsen.com

• Sponsor organisation

  Ipsen Innovation

• Research summary

  Osteosarcoma is a type of bone cancer that starts in cells that form bones. It is the most common bone cancer in children, adolescents, and young adults (AYA). The overall 5 year survival rate for osteosarcoma is around 60%.Current treatment includes chemotherapy and surgery. Therapeutic options are limited often with poor results. There is a great need for new therapies for the treatment of metastatic osteosarcoma in children and AYA.

  In osteosarcoma, several receptors get over-activated. which give very unusual signals in the body. Cabozantinib is a medication that can shut off many of these receptors.
  This study aims to understand how well maintenance cabozantinib works with best supportive care (BSC) to treat osteosarcoma when compared with BSC alone, after standard treatment. BSC is general medical care aimed at controlling symptoms and maximizing the quality of life without a specific active anticancer drug.

  Participants have a 50% chance of receiving cabozantinib initially. They can also switch to receiving it if the BSC is ineffective. Along with receiving a potential treatment, cabozantinib, participants will have medical assessments of their condition.
  Cabozantinib participants will receive cabozantinib tablet(s) once a day.
  Treatment will be given to the participants until the disease worsens, any safety issue occurs, the study doctor decides to stop the treatment or participant withdraws from the study.
  BSC—treatment will be given as per the study doctor’s assessment and the local guidelines. Participants on BSC treatment may switch to receive cabozantinib if their disease worsens.

  This study includes known risks, discomforts, or inconveniences, and risks that may not be foreseen. All known risks from previous studies will be monitored.

  Around 90 participants aged between 5 and 30 years at about 80 sites in 15 countries will take part in this study.
  This study is being sponsored by Ipsen Innovation.

• REC name

  East of Scotland Research Ethics Service REC 2

• REC reference

  24/ES/0022

• Date of REC Opinion

  11 Apr 2024

• REC opinion

  Further Information Unfavourable Opinion