TIP-12. A multicenter, retrospective-prospective real-world study: to evaluate the effectiveness and safety of Vorasidenib in Chinese patients with IDH1/2 mutant grade 2 astrocytoma or oligodendroglioma (VICTORIA): a trial-in-progress

Abstract The Phase 3 INDIGO study (NCT04164901) showed that Vorasidenib (VOR), a dual mutant isocitrate dehydrogenase 1 and 2 (mIDH1/2) inhibitor, significantly improved progression-free survival (PFS, HR 0.35) and delayed time to next intervention (TTNI, HR 0.20) over placebo in patients with Grade 2 mIDH1/2 diffuse glioma. However, robust clinical evidence in the Chinese population remains limited and urgently needed. While long follow-up is necessary to analyze conventional endpoints such PFS in mIDH1/2 gliomas. Tumor Growth Rate (TGR), measuring the percentage change in tumor volume, enables early indicator of treatment response in trials. Multiple studies, including INDIGO, have demonstrated the predictive nature of TGR for PFS. Therefore, a real-world study (RWS) evaluating the effectiveness and safety of VOR in Chinese mIDH1/2 glioma patients through 6-month TGR (6m-TGR) as an innovative primary endpoint was set up in the Chinese pilot zone Bo’ao, Hainan(NCT06969352). Twenty patients with Grade 2 mIDH1/2 glioma planning to receive VOR after surgery according to Bo’ao-approved indication will be prospectively enrolled (≥12 years; measurable non-enhancing disease; ≥1 surgery before VOR; no chemoradiotherapy before VOR) and compared with an external control group of 40 retrospectively identified patients with matched baseline characteristics and no VOR exposure. Safety data and MRI imaging will be collected routinely for one year. 6m-TGR, will serve as the primary endpoint. Other efficacy outcomes include real-world TTNI (rwTTNI) and real-world PFS (rwPFS). This represents the first prospective RWS to implement 6m-TGR as an early and sensitive endpoint for assessing the clinical benefit of VOR in Asian patients with glioma. The findings are expected to provide critical evidence to support regulatory decision-making and help accelerate the approval of VOR in China.