NCT07016282Active, not recruiting
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
Source: ClinicalTrials.gov · Last updated 2 Sept 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Study type
- Observational
- Enrollment
- 100 (estimated)
- First submitted
- 27 May 2025
- Start date
- 12 Jun 2025
- Primary completion date
- 30 Jun 2027
- Lead sponsor
- Mario Negri Institute for Pharmacological Research
- Diseases
- Polycystic kidney disease
- NCT number
- NCT07016282
What you commit to
- Study type: Observational
- Planned participants: 100
- From 12 Jun 2025 to the primary completion date, 30 Jun 2027
- 2 sites in 2 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Abnormal urinalysis suggestive of concomitant, clinically significant glomerular disease, and urinary tract lithiasis or infection
Other illness
- 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)
- Patients with major systemic disease
Other
- Patients unable to provide informed consent
- Pregnant, lactating, or potentially childbearing women without adequate contraception
Who the study is looking for
- 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
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Clinical Research Centre for Rare Diseases Aldo e Cele Daccò Coordinator of this site? Confirm its status | Ranica | Italy | Unknown status |
| Department of Global Public Health (GPH), Karolinska Institutet Coordinator of this site? Confirm its status | Stockholm | Sweden | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.