ADPKDADPKDNCT04338048
Autosomal Dominant Polycystic Kidney Disease (ADPKD) Study
An observational study of ADPKD, run by Children's Hospital of Philadelphia at 4 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- ADPKD
- How many people
- 300 participants
- Where
- 4 sitesUnited States
- Study dates
- 10 Oct 2019to 30 Oct 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients eligible are individuals diagnosed with ADPKD before the age of 18 years.
Who may be able to join
- Demonstration of ADPKD by clinical information, imaging studies, biopsy, autopsy, or genetic testing.
Who cannot join
- 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.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Enrolling by invitation
Cohen Children's Medical Center
New Hyde Park, New York, 11042
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146
Jasmine Jaber, MS, 267-425-5325, jaberj2@chop.edu
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55902
Christian Hanna, 507-721-4136, hanna.christian@mayo.edu
- Recruiting
Children's National Hospital
Washington D.C., District of Columbia, 20010
Ashima Gulati, 240-531-6619, agulati2@childrensnational.org
About this 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.
Read the detailed description
This study will ask the subject's permission to see past, current, and future medical information related to the disease. Some information that is collected, would be clinic notes, lab results, and physician consult reports. The subject will be asked to sign a release of medical information form to allow the study team access to medical information.This study does not require a clinic visit to the center.
When the research study receives the information, the research study team will be able to enter the medical data into the Hepato/Renal Fibrocystic Diseases clinical database.
There will be initial data entry in the database and follow up data entries lasting for the duration of this study or until the subject chooses to not participate in the study anymore. The study team will remove the subject's name or any other identifiable health information from the received records before entering medical data into the Hepato/Renal Fibrocystic Diseases clinical database.
- Conditions
- ADPKD
- Medical subject headings
- Polycystic Kidney, Autosomal Dominant
What is being measured
The primary outcome is the main question the study is designed to answer.
Database registry
Our research is designed to expand the currently limited knowledge about Autosomal Dominant Polycystic Kidney Diseases (ADPKD). Our specific goal is to create a registry of clinical information about this set of diseases.
This study has two parts, the clinical database (medical health information) and the educational part. In this study we want to build a registry with information about ADPKD to learn more about this disease. There will be an optional urine and/or blood collection.
Measured: up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2020
- Study start10 Oct 2019
- Primary completion30 Oct 2030 (estimated)
- Study completion30 Oct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ADPKDStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Collaborators
- National Institutes of Health (NIH)
- Responsible party
- Sponsor
- Investigators
- Lisa Guay-Woodford, MD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04338048
- Sponsor's protocol ID
- 23-021496
- Acronym
- ADPKD
- Status last verified
- Jun 2026
- First submitted
- 2 Jan 2020
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autosomal Dominant Polycystic Kidney Disease (ADPKD) Study
- ClinicalTrials.gov ID
- NCT04338048
- Acronym / sponsor ID
- ADPKD / 23-021496
- Conditions
- ADPKD
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Collaborators
- National Institutes of Health (NIH)
- Study start
- 10 Oct 2019 (actual)
- Primary completion
- 30 Oct 2030 (estimated)
- Study completion
- 30 Oct 2030 (estimated)
- First posted
- 8 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This study will ask the subject's permission to see past, current, and future medical information related to the disease. Some information that is collected, would be clinic notes, lab results, and physician consult reports. The subject will be asked to sign a release of medical information form to allow the study team access to medical information.This study does not require a clinic visit to the center.
When the research study receives the information, the research study team will be able to enter the medical data into the Hepato/Renal Fibrocystic Diseases clinical database.
There will be initial data entry in the database and follow up data entries lasting for the duration of this study or until the subject chooses to not participate in the study anymore. The study team will remove the subject's name or any other identifiable health information from the received records before entering medical data into the Hepato/Renal Fibrocystic Diseases clinical database.
Study design
- Observational model
- Other
- Time perspective
- Other
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients eligible are individuals diagnosed with ADPKD before the age of 18 years.
Key inclusion criteria (1 listed)
- Demonstration of ADPKD by clinical information, imaging studies, biopsy, autopsy, or genetic testing.
Key exclusion criteria (1 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Database registry
Time frame: up to 10 years
Our research is designed to expand the currently limited knowledge about Autosomal Dominant Polycystic Kidney Diseases (ADPKD). Our specific goal is to create a registry of clinical information about this set of diseases. This study has two parts, the clinical database (medical health information) and the educational part. In this study we want to build a registry with information about ADPKD to learn more about this disease. There will be an optional urine and/or blood collection.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2026.
- Related InfoLink provided by the sponsor.
Related trials for ADPKD
Active, not recruitingObservational study
Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Not yet recruitingEarly Phase 1
GBP-213 in Patients With Autosomal Dominant Polycystic Kidney Disease
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Safety, Tolerability and Pharmacokinetics of AZD1613 in Adults With Autosomal Dominant Polycystic Kidney Disease
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Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral Dose
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RecruitingPhase 2
ANCHOR Study: A Study to Assess the Safety and Efficacy of ABBV-CLS-628 in Adult Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Autosomal Dominant Polycystic Kidney Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Lisa Guay-Woodford, MD principal investigator
Children's Hospital of Philadelphia
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of ADPKD, run by Children's Hospital of Philadelphia at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jasmine Jaber, MS (267-425-5325, jaberj2@chop.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Database registry (time frame: up to 10 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jun 2026.