Vincent Ko
Founder & Executive Director KidneyFuture
Seminars
Thursday 20th August 2026
Reducing Recruitment Burden in Rare Congenital Kidney Disease Trials by Leveraging Natural History Data & External Control Arms to Minimize Placebo Requirements & Accelerate Patient Enrollment
9:00 am
- Leveraging natural history datasets, longitudinal patient registries, and real-world evidence to support external control cohorts in rare congenital kidney disease trials, including CAKUT
- Addressing recruitment challenges in small, heterogeneous, and pediatric populations by reducing reliance on large placebo arms and improving feasibility through smarter trial design
- Exploring how external control strategies, patient enrichment approaches, endpoint development, and public-private partnerships can strengthen trial readiness and accelerate future studies in congenital kidney disease
