Rahman R, Polley M-YC, Alder L et al (2023) Current drug development and trial designs in neuro-oncology: report from the first American Society of Clinical Oncology and Society for Neuro-Oncology clinical trials conference. Lancet Oncol 24(4):e161–e171. https://doi.org/10.1016/s1470-2045(23)00005-0

Article  PubMed  PubMed Central  CAS  Google Scholar 

Bagley SJ, Kothari S, Rahman R et al (2022) Glioblastoma clinical trials: current Landscape and opportunities for Improvement. Clin Cancer Res 28(4):594–602. https://doi.org/10.1158/1078-0432.CCR-21-2750.

Article  PubMed  PubMed Central  Google Scholar 

Tanguturi SK, Trippa L, Ramkissoon SH et al (2017) Leveraging molecular datasets for biomarker-based clinical trial design in glioblastoma. Neurooncology 19(7):908–917. https://doi.org/10.1093/neuonc/now312

Article  CAS  Google Scholar 

Hegi ME, Diserens A-C, Gorlia T et al (2005) MGMT Gene silencing and benefit from Temozolomide in Glioblastoma. N Engl J Med 352(10):997–1003. https://doi.org/10.1056/nejmoa043331

Article  PubMed  CAS  Google Scholar 

Hegi ME, Oppong FB, Perry JR et al (2024) No benefit from TMZ treatment in glioblastoma with truly unmethylated < i > MGMT promoter: reanalysis of the CE.6 and the pooled Nordic/NOA-08 trials in elderly glioblastoma patients. Neurooncology. https://doi.org/10.1093/neuonc/noae108

Article  Google Scholar 

Vogelbaum MA, Li G, Heimberger AB et al (2022) A window of opportunity to overcome therapeutic failure in Neuro-Oncology. Am Soc Clin Oncol Educ Book 42:1–8. https://doi.org/10.1200/EDBK_349175

Article  PubMed  Google Scholar 

Doroshow JH, Parchment RE (2008) Oncologic phase 0 trials incorporating clinical pharmacodynamics: from concept to patient. Clin Cancer Res 14(12):3658–3663. https://doi.org/10.1158/1078-0432.CCR-07-4562

Article  PubMed  PubMed Central  CAS  Google Scholar 

Mellinghoff IK, Lu M, Wen PY et al (2023) Vorasidenib and Ivosidenib in IDH1-mutant low-grade glioma: a randomized, perioperative phase 1 trial. Nat Med 29(3):615–622. https://doi.org/10.1038/s41591-022-02141-2

Article  PubMed  PubMed Central  CAS  Google Scholar 

Mellinghoff IK, Van Den Bent MJ, Blumenthal DT et al (2023) Vorasidenib in IDH1- or IDH2-Mutant low-Grade Glioma. N Engl J Med 389(7):589–601. https://doi.org/10.1056/nejmoa2304194

Article  PubMed  PubMed Central  CAS  Google Scholar 

Subbiah V, Kreitman RJ, Wainberg ZA et al (2023) Dabrafenib plus Trametinib in BRAFV600E-mutated rare cancers: the phase 2 ROAR trial. Nat Med 29(5):1103–1112. https://doi.org/10.1038/s41591-023-02321-8

Article  PubMed  CAS  Google Scholar 

Doz F, Van Tilburg CM, Geoerger B et al (2022) Efficacy and safety of larotrectinib in TRK fusion-positive primary central nervous system tumors. Neurooncology 24(6):997–1007. https://doi.org/10.1093/neuonc/noab274

Article  CAS  Google Scholar 

Rodón J, Damian S, Furqan M et al (2024) Pemigatinib in previously treated solid tumors with activating FGFR1–FGFR3 alterations: phase 2 FIGHT-207 basket trial. Nat Med 30(6):1645–1654. https://doi.org/10.1038/s41591-024-02934-7

Article  PubMed  CAS  Google Scholar 

Wen PY, Stein A, Van Den Bent M et al (2022) Dabrafenib plus Trametinib in patients with BRAFV600E-mutant low-grade and high-grade glioma (ROAR): a multicentre, open-label, single-arm, phase 2, basket trial. Lancet Oncol 23(1):53–64. https://doi.org/10.1016/s1470-2045(21)00578-7

Article  PubMed  CAS  Google Scholar 

Brastianos PK, Twohy E, Anders CK et al (2020) Alliance A071701: Genomically guided treatment trial in brain metastases. J Clin Oncol 38(15suppl):TPS2573–TPS2573. https://doi.org/10.1200/jco.2020.38.15_suppl.tps2573

Article  Google Scholar 

Kong BY, Sim H-W, Nowak AK et al (2021) LUMOS - Low and Intermediate Grade Glioma Umbrella Study of Molecular guided TherapieS at relapse: protocol for a pilot study. BMJ Open 11(12):e054075. https://doi.org/10.1136/bmjopen-2021-054075

Article  PubMed  PubMed Central  Google Scholar 

Wick W, Dettmer S, Berberich A et al (2019) N2M2 (NOA-20) phase I/II trial of molecularly matched targeted therapies plus radiotherapy in patients with newly diagnosed non-MGMT hypermethylated glioblastoma. Neurooncology 21(1):95–105. https://doi.org/10.1093/neuonc/noy161

Article  CAS  Google Scholar 

Brastianos PK, Twohy EL, Gerstner ER et al (2023) Alliance A071401: phase II trial of focal adhesion kinase inhibition in Meningiomas with somatic < i > NF2 mutations. J Clin Oncol 41(3):618–628. https://doi.org/10.1200/jco.21.02371

Article  PubMed  CAS  Google Scholar 

Rahman R, Trippa L, Lee EQ et al (2023) Inaugural results of the individualized screening trial of innovative glioblastoma therapy: a phase II platform trial for newly diagnosed Glioblastoma using bayesian adaptive randomization. J Clin Oncol 41(36):5524–5535. https://doi.org/10.1200/jco.23.00493

Article  PubMed  CAS  Google Scholar 

Alexander BM, Ba S, Berger MS et al (2018) Adaptive Global Innovative Learning Environment for Glioblastoma: GBM AGILE. https://doi.org/10.1158/1078-0432.CCR-17-0764

Plotkin SR, Yohay KH, Nghiemphu PL et al (2024) Brigatinib in < i > NF2 -Related schwannomatosis with progressive tumors. N Engl J Med 390(24):2284–2294. https://doi.org/10.1056/nejmoa2400985

Article  PubMed  CAS  Google Scholar 

Ellingson BM, Wen PY, Chang SM et al (2023) Objective response rate targets for recurrent glioblastoma clinical trials based on the historic association between objective response rate and median overall survival. Neurooncology 25(6):1017–1028. https://doi.org/10.1093/neuonc/noad002

Article  CAS  Google Scholar 

Han K, Ren M, Wick W et al (2014) Progression-free survival as a surrogate endpoint for overall survival in glioblastoma: a literature-based meta-analysis from 91 trials. Neurooncology 16(5):696–706. https://doi.org/10.1093/neuonc/not236

Article  Google Scholar 

Wen PY, Macdonald DR, Reardon Da et al (2010) Updated response assessment criteria for high-grade gliomas: response assessment in neuro-oncology working group. J Clin Oncol 28(11):1963–1972. https://doi.org/10.1200/JCO.2009.26.3541

Article  PubMed  Google Scholar 

Ellingson BM, Wen PY, Cloughesy TF (2017) Modified Criteria for Radiographic Response Assessment in Glioblastoma clinical trials. Neurotherapeutics 14(2):307–320. https://doi.org/10.1007/s13311-016-0507-6

Article  PubMed  PubMed Central  Google Scholar 

Youssef G, Rahman R, Bay C et al (2023) Evaluation of Standard Response Assessment in Neuro-Oncology, Modified Response Assessment in Neuro-Oncology, and Immunotherapy Response Assessment in Neuro-Oncology in newly diagnosed and recurrent glioblastoma. J Clin Oncol 41(17):3160–3171. https://doi.org/10.1200/jco.22.01579

Article  PubMed  CAS  Google Scholar 

Friedman HS, Prados MD, Wen PY et al (2009) Bevacizumab alone and in combination with irinotecan in recurrent glioblastoma. J Clin Oncol 27(28):4733–4740. https://doi.org/10.1200/JCO.2008.19.8721

Article  PubMed  CAS  Google Scholar 

Kilburn LB, Khuong-Quang D-A, Hansford JR et al (2024) The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial. Nat Med 30(1):207–217. https://doi.org/10.1038/s41591-023-02668-y

Article  PubMed  CAS  Google Scholar 

Scheepens JCC, Taphoorn MJB, Koekkoek JAF (2024) Patient-reported outcomes in neuro-oncology. Curr Opin Oncol. https://doi.org/10.1097/cco.0000000000001078

Article  PubMed  Google Scholar 

Fangusaro J, Avery RA, Fisher MJ et al (2024) Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma Clinical Trials: An FDA Educational Symposium. Clinical Cancer Research.;30(11):2303–2308. https://doi.org/10.1158/1078-0432.ccr-23-3386

Ellingson BM, Kim GHJ, Brown M et al (2022) Volumetric measurements are preferred in the evaluation of mutant IDH inhibition in non-enhancing diffuse gliomas: evidence from a phase I trial of ivosidenib. Neurooncology 24(5):770–778. https://doi.org/10.1093/neuonc/noab256

Article  CAS  Google Scholar 

Grossman SA, Schreck KC, Ballman K, Alexander B (2017) Point/counterpoint: randomized versus single-arm phase II clinical trials for patients with newly diagnosed glioblastoma. Neurooncology 19(4):469–474.