Optimising combination therapy for paediatric, adolescent and young adult patients with non-rhabdomyosarcoma soft tissue sarcomas: The OCTOPUS platform trial

Few innovative treatments were developed for patients with non-rhabdomyosarcoma soft tissue sarcomas (NRSTS) in the past decades. The paper describes the OCTOPUS project (Optimising Combination Therapy fOr Paediatric, adolescent and yoUng adult patients with non-rhabdomyosarcoma soft tissue Sarcomas), a master protocol and includes an adaptive platform trial comprising different sub-trials, a real-world data registry, translational studies, and overarching study questions assessing local therapy issues and patient reported outcome measures (PROMs). The OCTOPUS consortium will provide an operational framework including a legal consortium structure, a network of national coordinating centres (NCCs) and sites within the EpSSG (European paediatric Soft tissue sarcoma Study Group) and ITCC (Innovative Therapies for Children and adolescents with Cancer) network. The overarching aim of the platform is to improve outcome and quality of life for patients with NRSTS by providing access to innovative treatments. Every sub-trial will have a unique design, tailored to the needs of the patients, the characteristics of the disease, and the stage of development of the experimental compound(s). Depending on the medical need in a specific patient population and the expected activity of a compound (or a combination), the innovative treatment(s) will be offered to patients with relapsed/refractory disease or placed in frontline treatment when appropriate.