Innovative clinical trial designs for glioblastoma (GBM) are crucial for accelerating drug discovery. Phase 0, window of opportunity, and adaptive designs have emerged as promising approaches, though their advanced methodologies and biostatistics are not yet widely understood. This review focuses on these designs, tailored specifically for physicians.
Recent developments have seen the implementation of Phase 0, window of opportunity, and adaptive trials in GBM research. These innovative designs aim to eliminate ineffective therapies earlier in the drug development process, thereby enhancing trial efficiency. Notably, two ongoing adaptive platform trials are making significant strides: the GBM Adaptive Global Innovative Learning Environment (GBM AGILE) and the INdividualized Screening trial of Innovative GBM Therapy (INSIGhT).
The future of GBM clinical trials is expected to increasingly incorporate Phase 0, window of opportunity, and adaptive Phase I-III studies. The success of these trials will largely depend on the continued collaboration between physicians and biostatisticians, ensuring the effective implementation of these advanced trial designs.