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NCT01401998 · ClinicalTrials.gov registry record

ARPKD Database Study

A clinical trial of Bardet-Biedl Syndrome and Autosomal Recessive Polycystic Kidney Disease, sponsored by Children's Hospital of Philadelphia.

Recruiting
Registry status
200
Enrollment target
6
Study locations

NCT01401998 is a study of Bardet-Biedl Syndrome and Autosomal Recessive Polycystic Kidney Disease that is actively recruiting participants, run by Children's Hospital of Philadelphia. The registered enrollment target is 200 participants. The trial reports 6 study locations across 6 states.

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The verdict

NCT01401998, a study of Bardet-Biedl Syndrome and Autosomal Recessive Polycystic Kidney Disease, is actively recruiting participants, sponsored by Children's Hospital of Philadelphia.

RECRUITING
Registry status
200 participants
Enrollment target
6
Study locations

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

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

FieldValue
Enrollment Target 200 participants
Start Date 2011-06
Est. Completion 2030-12

Sponsor

Children's Hospital of Philadelphia

431 total trials

What the Registry Record Tells You About NCT01401998

The ClinicalTrials.gov registry entry for NCT01401998 describes a study currently listed as recruiting, categorized as an unspecified phase. 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 431 total studies on file at ClinicalTrials.gov.

The record links to 10 conditions, with Bardet-Biedl Syndrome appearing as the primary indexed condition, and to 0 interventions.

NCT01401998 reports 6 study locations spanning 6 distinct geographic areas - top geographies include District of Columbia, Georgia, Massachusetts.

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: Bardet-Biedl Syndrome, Autosomal Recessive Polycystic Kidney Disease, Congenital Hepatic Fibrosis, Hepato/Renal Fibrocystic Disease, Joubert Syndrome.

Who is sponsoring clinical trial NCT01401998?

This trial is sponsored by Children's Hospital of Philadelphia, which has 431 total clinical trials registered on ClinicalTrials.gov.

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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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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