The Future Glioblastoma Clinical Trials Landscape: Early Phase 0, Window of Opportunity, and Adaptive Phase I–III Studies

PURPOSE OF REVIEW: Innovative clinical trial designs for glioblastoma (GBM) are needed to expedite drug discovery. Phase 0, window of opportunity, and adaptive designs have been proposed, but their advanced methodologies and underlying biostatistics are not widely known. This review summarizes phase 0, window of opportunity, and adaptive phase I–III clinical trial designs in GBM tailored to physicians. RECENT FINDINGS: Phase 0, window of opportunity, and adaptive trials are now being implemented for GBM. These trials can remove ineffective therapies earlier during drug development and improve trial efficiency. There are two ongoing adaptive platform trials: GBM Adaptive Global Innovative Learning Environment (GBM AGILE) and the INdividualized Screening trial of Innovative GBM Therapy (INSIGhT). SUMMARY: The future clinical trials landscape in GBM will increasingly involve phase 0, window of opportunity, and adaptive phase I–III studies. Continued collaboration between physicians and biostatisticians will be critical for implementing these trial designs.