Schoot RA , Casanova M, van Bindsbergen K, Brennan B, Breunis B, Di Carlo D, Corradini N, Davila Fajardo R, Ferrari Bravo W, Guillén Burrieza G, Grootenhuis M, Hovsepyan S, Huitema ADR, Meister MT, Metayer L, van Noesel MM, Orbach D, Riedl D, van Scheltinga ST, van Tinteren H, Valerio D, Scobie N, Ferrari A
Tumori. 2026 Jul 30:3008916261456574. doi: 10.1177/03008916261456574. Online ahead of print.
This paper, authored by members of our EpSSG NRSTS Committee, describes the upcoming OCTOPUS study, which is set to launch within our community in the coming months. OCTOPUS stands for Optimising Combination Therapy fOr Paediatric, adolescent and yoUng adult patients with non-rhabdomyosarcoma soft tissue Sarcomas. The article outlines the challenges faced over recent years in developing protocols for NRSTS and gaining access to novel treatments, presenting OCTOPUS as a viable solution to these hurdles.
Designed as a master protocol, OCTOPUS 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). Furthermore, the paper explains how the OCTOPUS consortium will provide an operational framework, including a legal consortium structure and a network of national coordinating centres (NCCs) and sites within both the EpSSG and ITCC networks.
The authors highlight that OCTOPUS will act as a dynamic, adaptive platform capable of running multiple interventional sub-trials across rare disease entities, either concurrently or sequentially. Every sub-trial will feature a unique design tailored to patient needs, disease characteristics, and the developmental stage of the experimental compound(s). Depending on the specific medical needs and the expected activity of a compound (or combination), innovative treatments will be offered to patients with relapsed/refractory disease or integrated into frontline treatment where appropriate. The first sub-trials to launch will focus on desmoid fibromatosis, malignant rhabdoid tumours, and desmoplastic small round cell tumours.
OCTOPUS marks the dawn of a new generation of NRSTS trials for our community. We believe this project accomplishes far more than simply testing novel pharmaceutical compounds: it establishes a dynamic, sustainable, and truly international research ecosystem designed to ensure that even the rarest tumour types are no longer left behind in the era of precision medicine.
Designed as a master protocol, OCTOPUS 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). Furthermore, the paper explains how the OCTOPUS consortium will provide an operational framework, including a legal consortium structure and a network of national coordinating centres (NCCs) and sites within both the EpSSG and ITCC networks.
The authors highlight that OCTOPUS will act as a dynamic, adaptive platform capable of running multiple interventional sub-trials across rare disease entities, either concurrently or sequentially. Every sub-trial will feature a unique design tailored to patient needs, disease characteristics, and the developmental stage of the experimental compound(s). Depending on the specific medical needs and the expected activity of a compound (or combination), innovative treatments will be offered to patients with relapsed/refractory disease or integrated into frontline treatment where appropriate. The first sub-trials to launch will focus on desmoid fibromatosis, malignant rhabdoid tumours, and desmoplastic small round cell tumours.
OCTOPUS marks the dawn of a new generation of NRSTS trials for our community. We believe this project accomplishes far more than simply testing novel pharmaceutical compounds: it establishes a dynamic, sustainable, and truly international research ecosystem designed to ensure that even the rarest tumour types are no longer left behind in the era of precision medicine.
