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ARPKD Database Study
NCT01401998 · View on ClinicalTrials.gov ↗
Study Summary
Hepato-renal fibrocystic diseases (HRFD) is a term developed that encompasses rare diseases such as Autosomal Recessive Polycystic Kidney Disease (ARPKD), and other diseases with common features (Joubert syndrome, Bardet Biedl syndrome, Meckel-Gruber syndrome, congenital hepatic fibrosis (CHF), Caroli syndrome (CS), polycystic liver disease, oro-facial-digital syndrome, nephronophithisis (NPHP), and glomerulocystic Kidney Disease). The lack of enough routinely available resources for these diseases to be well diagnosed and treated, would be best resolved by coordinated case accrual and sharing of clinical data and bio-specimens (DNA and tissues) among participating institutions, thereby leading to the centralization and sharing of clinical and genetic information, as well as bio-materials, providing an important engine for more rapid research progress and community understanding through the creation of research networks. This study aims to build a registry of a clinical database (medical health information), a mutational database (genetic information) and an educational resource about HRFD to eventually provide information about these diseases to families, physicians and genetic counselors via our existing HIPAA- approved study website. Goals for the Core A: The Hepato/Renal Fibrocystic Diseases Translational Resource are: 1. \- Clinical Database: • Expand our comprehensive Clinical Database to include information from all patients who meet the inclusion criteria for hepato/renal fibrocystic diseases. 2. \- Mutational Database: * Test children with ARPKD and other hepato/renal fibrocystic disease to identify genetic mutations, establish a DNA bank for patients with hepato/renal fibrocystic diseases and develop a Mutational Database. This Database will be capable of linking clinical and mutational information via a unique identifier in a searchable format to facilitate genetic research (e.g. genotype-phenotype correlations, new disease gene studies, and m
Conditions Studied
Study Locations (6)
District of Columbia
- Children's National Hospital — Washington D.C.
Georgia
- Emory — Atlanta
Massachusetts
- Boston Children's — Boston
Ohio
- Cincinnati Children's — Cincinnati
Pennsylvania
- Children's Hospital of Philadelphia — Philadelphia
Utah
- University of Utah — Salt Lake City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 200 participants |
| Start Date | 2011-06 |
| Est. Completion | 2030-12 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01401998
The ClinicalTrials.gov registry entry for NCT01401998 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 200 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Children's Hospital of Philadelphia, which has 492 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 10 conditions, with Autosomal Recessive Polycystic Kidney Disease appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT01401998 reports 6 study locations spanning 6 distinct geographic areas — top geographies include District of Columbia, Georgia, Massachusetts. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT01401998 about?
NCT01401998 is a clinical study titled "ARPKD Database Study". Hepato-renal fibrocystic diseases (HRFD) is a term developed that encompasses rare diseases such as Autosomal Recessive Polycystic Kidney Disease (ARPKD), and other diseases with common features (Joubert syndrome, Bardet Biedl syndrome, Meckel-Gruber syndrome, congenital hepatic fibrosis (CHF), Caro...
What is the current status of trial NCT01401998?
This trial is currently recruiting. The enrollment target is 200 participants. The study started on 2011-06. Estimated completion is 2030-12.
What conditions does trial NCT01401998 study?
This clinical trial studies the following conditions: Autosomal Recessive Polycystic Kidney Disease, Congenital Hepatic Fibrosis, Hepato/Renal Fibrocystic Disease, Joubert Syndrome, Bardet Biedl Syndrome. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT01401998?
This trial is sponsored by Children's Hospital of Philadelphia, which has 492 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT01401998 being conducted?
This trial has 6 study locations across District of Columbia, Georgia, Massachusetts, Ohio, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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