Active — Not RecruitingNot Applicable

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

Italy, Sweden100 participantsStarted 2025-06-12

Plain-language summary

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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Adult (\>18 years) men and women with ADPKD according to Ravine criteria25 * Estimated glomerular filtration rate (eGFR) between 15 and 40 mL/min/1.73 m2 (CKD stage: G3b-G4) or higher (CKD stage: G1-G3a), as calculated by the Modification of Diet in Renal Disease study four variables equation Exclusion Criteria: * confounding factors that could affect renal function loss independent of kidney growth and treatment allocation (i.e., diabetes mellitus, urinary protein excretion rate \>3 g/24 h) * Abnormal urinalysis suggestive of concomitant, clinically significant glomerular disease, and urinary tract lithiasis or infection * Patients with major systemic disease * Patients unable to provide informed consent * Pregnant, lactating, or potentially childbearing women without adequate contraception

What they're measuring

Changes in total kidney volume (TKV)

Timeframe: At baseline, and immediately after data generation procedure.

Trial details

NCT IDNCT07016282

SponsorMario Negri Institute for Pharmacological Research

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2027-06