Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: Trade Data, Destination Markets, and Economic Impact Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: Trade Data, Destination Markets, and Economic Impact
Clinical Trials in Italy / NCT07016282
Active, not recruiting Observational

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

NCT07016282 · tracked via the Priya Life Science Italy tracker
Sponsor
Mario Negri Institute for Pharmacological Research
Phase
Observational
Started
2025-06-12
Last updated
2025-09-02

Condition(s) studied

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Study 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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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

Primary outcome measure(s)

  • Changes in total kidney volume (TKV) — At baseline, and immediately after data generation procedure.
    Changes in TKV in mL.

Trial sites (2)

FacilityCityRegionStatus
Clinical Research Centre for Rare Diseases Aldo e Cele Daccò Ranica BG
Department of Global Public Health (GPH), Karolinska Institutet Stockholm Sweden

More Mario Negri Institute for Pharmacological Research trials in Italy

Evaluation of the Quality of Care in the Emergency Department by Studying the Appropriateness of Admissions of Patients Accessing the Emergency Department (ASST Papa Giovanni XXIII) Active, not recruiting
Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases Recruiting
Severe Acquired Brain Injuries: Prognostic Factors and Quality of Care Recruiting
Health-care Quality in Semi-intensive Care Units Recruiting
Artificial Intelligence-based Image Processing Methods to Advance the Characterization of Polycystic Kidney Disease Active, not recruiting

Other trials for the same condition

RADIOLOGICAL AND CLINICAL EVALUATION OF RENAL EMBOLIZATION USING EVOH IN DIALYSIS PATIENTS WITH AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE: A PROSPECTIVE LONGITUDINAL OBSERVATIONAL STUDY Recruiting
Artificial Intelligence-based Image Processing Methods to Advance the Characterization of Polycystic Kidney Disease Active, not recruiting
Robotic vs Open Nephrectomy for ADPKD Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07016282 on ClinicalTrials.gov ↗ ← All trials in Italy