Recent Status of Phase I Clinical Trials for Brain Tumors: A Regulatory Science Study of Exploratory Efficacy Endpoints.

Shinya WatanabeTakahiro NonakaMakoto MaedaMasanobu YamadaNarushi SugiiKoichi HashimotoShingo TakanoTomoyoshi KoyanagiYoshihiro ArakawaEiichi Ishikawa

Published in: Therapeutic innovation & regulatory science (2024)

Recent Phase I trials included efficacy endpoints as SEs, with ORR, PFS, or OS included in ~ 50% trials and DOR or DCR in ~ 25%. No established criteria exist for imaging evaluation of BTs. Phase I trials involving mixed solid tumor cohorts revealed challenges in designing methods to assess the exploratory efficacy of BTs.

Keyphrases

clinical trial
public health
high resolution
randomized controlled trial
transcription factor
fluorescence imaging
study protocol