NF2-related Schwannomatosis

This project aims to improve outpatient departments by working with patients and staff to make these spaces more inclusive for people with sensory challenges (it focuses on people with nerve tumour conditions as they are more likely to have sensory challenges). The project uses a well-established method called experience-based co-design (EBCD), which supports patients and staff to work together. It will run over 4 years.

This is an adaptive platform-basket trial that aims to evaluate the safety and efficacy of multiple novel agents and combination therapies in patients with NF2-related schwannomatosis (NF2-SWN). The study employs a basket design to assess treatment responses across four tumor types commonly associated with NF2-SWN: vestibular schwannomas, non-vestibular schwannomas, meningiomas, and ependymomas. A shared natural history observational cohort, receiving routine clinical follow-up without investigational treatment, serves as a common control for all substudies. The adaptive platform enables the dynamic addition or closure of substudies based on interim analyses, thereby optimizing trial efficiency. Eligible patients who meet the master protocol criteria and satisfy substudy-specific safety requirements will be assigned to receive the corresponding intervention. Currently open substudies include: * Substudy A: Selumetinib * Substudy B: Luvometinib plus Serplulimab

The goal of this clinical trial is to evaluate the efficacy and safety of the MEK1/2 inhibitor selumetinib in treating patients with neurofibromatosis type 2-related schwannomatosis (NF2-SWN), including both adults and children with inoperable or progressive tumors.

This is an investigator-initiated, exploratory, multicenter, open-label, single-arm clinical trial and a substudy of the Platform Research for Innovative Medicines in NF2-SWN (PRIME-NF2). The study aims to evaluate the safety, tolerability, and preliminary efficacy of luvometinib in combination with serplulimab in patients with NF2-related schwannomatosis (NF2-SWN) with progressive tumors.