Understanding Autosomal Dominant Polycystic Kidney Disease in Children
Official title: Autosomal Dominant Polycystic Kidney Disease (ADPKD) Study
Run by Children's Hospital of Philadelphia · NCT04338048 · Data updated 2026-11-12
This study is looking to gather medical information about how Autosomal Dominant Polycystic Kidney Disease affects children to help improve care and resources for families.
Quick facts
| Study type | Observational |
|---|---|
| Ages | Any age to 18 Years |
| Sex | All |
| Enrollment | 300 (estimated) |
| Sponsor | Children's Hospital of Philadelphia Academic / other |
| Locations | 4 sites (Washington D.C., District of Columbia and 3 other locations) |
| Status | Recruiting |
| Started | October 10, 2019 |
| Main results expected | October 30, 2030 |
| Study ends | October 30, 2030 |
| Registry record updated | June 15, 2026 |
| Trial ID | NCT04338048 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include individuals diagnosed with Autosomal Dominant Polycystic Kidney Disease through clinical information, imaging, or genetic testing.
✕Probably not a fit
Patients with Autosomal Recessive Polycystic Kidney Disease or significant urinary tract malformations may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study aims to gather comprehensive medical information on Autosomal Dominant Polycystic Kidney Disease (ADPKD) and related hepato/renal fibrocystic diseases, particularly focusing on the disease's progression in children. Participants will provide consent for the study team to access their past, current, and future medical records, which will be anonymized and entered into a specialized clinical database. The study seeks to fill the gap in evidence-based guidelines for managing ADPKD in pediatric patients and expand educational resources available online. No clinic visits are required for participation, making it accessible for families.
Why it matters
Potential benefit: If successful, this study could lead to improved management strategies and resources for children diagnosed with ADPKD.
How similar studies have gone: While there is limited data on similar studies focusing on pediatric ADPKD, the approach of gathering comprehensive medical histories has shown promise in other chronic conditions.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Demonstration of ADPKD by clinical information, imaging studies, biopsy, autopsy, or genetic testing. Exclusion Criteria: * 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.
Where it's running
-
Children's National Hospital
Washington D.C., District of Columbia, United States Recruiting -
Mayo Clinic
Rochester, Minnesota, United States Recruiting -
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States Recruiting -
Cohen Children's Medical Center
New Hyde Park, New York, United States Enrolling by invitation
Study contacts
- Principal investigator: Lisa Guay-Woodford, MD — Children's Hospital of Philadelphia
- Study coordinator: Jasmine Jaber, MS
- Email: jaberj2@chop.edu
- Phone: 267-425-5325
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Mapping sensory pain patterns in ADPKD
This project will test whether detailed sensory testing and short questionnaires can identify characteristic pain patterns in people aged 12 and older with ADPKD compared with matched healthy volunteers.
-
Database analysis of patients with Autosomal Dominant Polycystic Kidney Disease
This study is trying to gather and track health information from people with Autosomal Dominant Polycystic Kidney Disease to see how different treatments affect their kidney function and cyst growth over time.
-
Evaluating the role of genetics in kidney disease progression
This study is trying to see how genetics affects the worsening of kidney diseases like acute kidney injury and chronic kidney disease in people, especially those with high blood pressure.
-
Hydrochlorothiazide added to tolvaptan to protect kidneys and improve quality of life in ADPKD
This trial tests whether adding low-dose hydrochlorothiazide to adults with ADPKD who are already taking tolaptan slows kidney decline and reduces side effects like excessive urination.
-
Imaging kidney sodium levels using MRI
This study is testing if a special MRI can show differences in sodium levels in the kidneys of people with various kidney diseases to help improve their treatment.
-
VX-407 pharmacokinetics after a single dose in adults with ADPKD
This study tests how VX-407 is absorbed, distributed, metabolized, and excreted after a one-time oral dose in adults with polycystic kidney disease.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.