First-in-human Phase 1 dose escalation with primary focus on safety and tolerability; anti-tumor activity is secondary and minimal (2.5% ORR), consistent with early-phase exploratory work.
Single-arm neoadjuvant study with comprehensive molecular profiling showing early tumour and microenvironmental changes, but limited by small sample size, lack of control arm, and surrogate endpoints rather than hard clinical outcomes.
Single-centre feasibility study of organoid drug testing in a small sample with uncontrolled clinical outcomes; demonstrates proof-of-concept but lacks comparison group and prospective validation.
A pilot clinical trial with critically limited enrollment (N=3) that confirms safety and feasibility but cannot establish efficacy; accompanied by mechanistic preclinical work in a mouse model.