Autosomal Dominant Polycystic Kidney Disease (ADPKD) Study

Sponsor
Children's National Research Institute (Other)
Overall Status
Recruiting
CT.gov ID
NCT04338048
Collaborator
(none)
300
1
72.7
4.1

Study Details

Study Description

Brief 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. 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.

Condition or Disease Intervention/Treatment Phase

    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

    Study Type:
    Observational
    Anticipated Enrollment :
    300 participants
    Observational Model:
    Other
    Time Perspective:
    Other
    Official Title:
    Autosomal Dominant Polycystic Kidney Disease (ADPKD) Study
    Actual Study Start Date :
    Oct 10, 2019
    Anticipated Primary Completion Date :
    Oct 30, 2025
    Anticipated Study Completion Date :
    Oct 30, 2025

    Outcome Measures

    Primary Outcome Measures

    1. Medical Record Review [up to 10 years]

      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.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Children's National Hospital Washington District of Columbia United States 20010

    Sponsors and Collaborators

    • Children's National Research Institute

    Investigators

    • Principal Investigator: Lisa Guay-Woodford, MD, Children's National Research Institute

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Lisa M. Guay-Woodford, Principal Investigator, Children's National Research Institute
    ClinicalTrials.gov Identifier:
    NCT04338048
    Other Study ID Numbers:
    • Pro00012541
    First Posted:
    Apr 8, 2020
    Last Update Posted:
    Jul 27, 2021
    Last Verified:
    Jul 1, 2021
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Jul 27, 2021