P1, N=3, Completed, City of Hope Medical Center | Active, not recruiting --> Completed | Trial completion date: May 2027 --> Mar 2026 | Trial primary completion date: May 2027 --> Mar 2026

The IDH-mutant inhibitor vorasidenib has been demonstrated to prolong progression-free survival in grade 2 IDH-mutant glioma; however, there is a lack of evidence in patients younger than 18...Craniopharyngioma is a rare CNS tumor in the AYA population, and BRAFV600E mutations in papillary craniopharyngioma represent a targetable alteration. Solutions to improving the care of AYA should include appropriate representation in clinical trials and specialized care by experienced clinicians.

In conclusion, vorasidenib was generally safe and well tolerated, and plasma exposures were generally similar between Japanese and non-Asian participants following single oral 10- and 50-mg vorasidenib doses. These results enabled vorasidenib clinical development in Japan and supported inclusion of Japanese sites in subsequent vorasidenib clinical trials without dose adjustments.

This study identifies PIK3CG, PRKCD, and TRIM22 as potential biomarkers and therapeutic targets in IDH-wildtype GBM. Their paradoxical association with poor survival and immune activation may inform personalized treatment strategies that combine conventional chemotherapy with immune-based therapies. While our findings are robust across both mixed and IDH-wildtype-focused cohorts, further mechanistic validation is warranted.

P=N/A, N=90, Recruiting, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

The striking reductions in tumor 2HG and tCho levels early following initiation of targeted therapy using an IDH inhibitor suggest that MRS may provide an important tool for monitoring treatment response of lower-grade gliomas.

Therapeutic advances are highlighted, with particular emphasis on brain-penetrant IDH inhibition (vorasidenib) and on emerging strategies including vaccines, checkpoint combinations, epigenetic modulation, metabolic and microenvironment targeting, and novel delivery platforms...Finally, future directions in trial design, survivorship-oriented endpoints, and biomarker-driven monitoring are outlined. A trajectory-based paradigm is emphasized in which neurocognitive preservation, time to radiotherapy or chemotherapy, and patient-reported outcomes are prioritized while durable disease control is pursued across decades-long survivorship.

This work delivers multidisciplinary consensus recommendations to standardize care in IDH-mutant grade 2 gliomas, integrating advances in molecular diagnostics, imaging, and therapeutics. It also identifies key knowledge gaps and clinical uncertainties, underscoring the critical need for ongoing research and expert collaboration to continually refine personalized management approaches and improve patient outcomes.

P1, N=18, Recruiting, Memorial Sloan Kettering Cancer Center | N=36 --> 18

P2, N=35, Recruiting, City of Hope Medical Center | Suspended --> Recruiting

P=N/A, N=10, Not yet recruiting, Huashan Hospital

P1, N=60, Active, not recruiting, Institut de Recherches Internationales Servier | Recruiting --> Active, not recruiting