Simulated and Synthetic Health Data: Improving Clinical Research on Rare Diseases. A Real-World Data Simulation of Autosomal Dominant Polycystic Kidney Disease (ADPKD) Trials. A Retrospective, Observational Study

This is a no-profit, retrospective observational study involving real-world data (RWD), retrieved from ADPKD-related electronic health records stored at Mario Negri Institute IRCCS. RWD will be used to generate simulated and synthetic datasets, using AI tools. RWD and generated data (GD) will be used to conduct three virtual RCTs, which main outcome is change in Total Kidney Volume (TKV). Statistical tests will be performed to assess quality and privacy preservation of GD compared with RWD. GD will be also evaluated in exploratory sample size estimations.

NCT Number	NCT07016282
Lead Sponsor	Mario Negri Institute for Pharmacological Research
Conditions	Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Enrollment	100 participants
Start Date	2025-06-12
Primary Completion	2027-06 (estimated)
Study Completion	2027-06 (estimated)
Updated on ClinicalTrials.gov	2025-09-02