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Clinical Investigations Into the Kidney and Liver Disease in Autosomal Recessive Polycystic Kidney Disease/Congenital Hepatic Fibrosis and Other Ciliopathies

This study is currently Recruiting

March 2011 By National Institutes of Health Clinical Center (CC)

First Recieved on September 10, 2003

Last Updated on April 22, 2011

Sponsor: National Human Genome Research Institute (NHGRI)
Collaborators:
Information provided by: National Institutes of Health Clinical Center (CC)
Identifier: NCT00068224

Purpose

This study will evaluate patients with autosomal recessive polycystic kidney disease (ARPKD) and congenital hepatic fibrosis (CHF) and other related disorders (ciliopathies). People with ARPKD develop kidney cysts and eventually kidney failure, symptoms may include hypertension (high blood pressure), poor growth, and urinary infections. CHF is a specific type of liver disease associated with ARPKD. It involves fibrosis, or scarring, of the liver, which can lead to life-threatening complications, including internal bleeding of enlarged blood vessels called varices in the esophagus (food pipe). The goal of the study is to better understand the medical complications of these disorders and identify characteristics that can help in the design of new treatments. ...

Study Type: Observational
Study Design: N/A

Eligibility

Ages Eligible for Study:6 Months
Genders Eligible for Study:Both
Accepts Healthy Volunteers:No
Criteria

- INCLUSION CRITERIA: This protocol will enroll children and adults who carry a clinical diagnosis of ARPKD, CHF, JSRD, BBS, OFD or AS and who has either PKD/NP spectrum of changes in the kidneys or CHF/Caroli's syndrome of the liver. This might rarely include adults who are unable to give informed consent. Among patients who have received a kidney or liver allograft, those with stable graft function and without severe transplant-related complications are eligible for enrollment. Patients and their parents/legal guardians must be willing to come to the NIH Clinical Center for admission annually. EXCLUSION CRITERIA: Infants under 6 months of age. Medically fragile patients who require frequent hospitalizations due to complications of end-stage renal disease (uncontrolled hypertension, severe electrolyte imbalances) or hepatic disease (current variceal bleeding, overt encephalopathy, intractable recurrent cholangitis).

Investigators

Locations

  • National Institutes of Health Clinical Center, 9000 Rockville Pike

    Bethesda, Maryland 20892 United States

Conditions related to this trial:

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