Study following children and young people with a genetic kidney cyst disease
Observes how polycystic kidney disease, inherited from a parent, progresses in people up to 18.
Plain-language title generated from the registry record
Registry title: Autosomal Dominant Polycystic Kidney Disease (ADPKD) Study
Key facts
- Where
- New Hyde Park, Philadelphia, Rochester, and Washington D.C., United StatesSee the 4 sites
Where in United States
- Cohen Children's Medical CenterNew Hyde Park, United StatesEnrolling by invitation
- Children's Hospital of PhiladelphiaPhiladelphia, United StatesTaking people
- Mayo ClinicRochester, United StatesTaking people
- Children's National HospitalWashington D.C., United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Children's Hospital of Philadelphia
- Trial code
- NCT04338048
- Record updated
- 15 June 2026
- Original description
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common genetic cause of renal failure. For several decades, ADPKD was regarded as an adult-onset disease. In the last decade, it has become more widely appreciated that the disease course begins in childhood. However, evidence-based guidelines on how to manage and approach children diagnosed with or at-risk for of ADPKD are lacking. Overall, there is insufficient data on the clinical course during childhood. The study intends to get more information on Autosomal Dominant Polycystic Kidney Disease (ADPKD) and other hepato/renal fibrocystic diseases. Additionally, the study intends to expand web-based resources so anyone can learn about ADPKD or other hepato/renal fibrocystic diseases. Individuals diagnosed with the dominant form of a hepato/renal fibrocystic condition are invited to be in the study.Read full original descriptionShow less
Autosomal Dominant Polycystic Kidney Disease (ADPKD) Study
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common genetic cause of renal failure. For several decades, ADPKD was regarded as an adult-onset disease. In the last decade, it has become more widely appreciated that the disease course begins in childhood. However, evidence-based guidelines on how to manage and approach children diagnosed with or at-risk for of ADPKD are lacking. Overall, there is insufficient data on the clinical course during childhood. The study intends to get more information on Autosomal Dominant Polycystic Kidney Disease (ADPKD) and other hepato/renal fibrocystic diseases. Additionally, the study intends to expand web-based resources so anyone can learn about ADPKD or other hepato/renal fibrocystic diseases. Individuals diagnosed with the dominant form of a hepato/renal fibrocystic condition are invited to be in the study.
Inclusion: Demonstration of ADPKD by clinical information, imaging studies, biopsy, autopsy, or genetic testing.
Exclusion: Patients with Autosomal Recessive Polycystic Kidney disease (ARPKD), urinary tract malformations or major congenital anomalies of other systems suggesting a diagnosis other than recessive hepato-renal fibrocystic diseases.