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Positional Cloning of the Gene(s) Responsible for Alagille Syndrome
This study has been completed.
Sponsored by: National Human Genome Research Institute (NHGRI)
Information provided by: National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00001642
  Purpose

The goal of the project is to identify and clone the gene(s) responsible for the Alagille Syndrome (AGS) by a positional cloning approach. The first step towards this goal is to define the smallest genomic candidate region for AGS at 20p12 and to begin to identify genes within this region which are, by definition, candidate genes for the disease. In a collaborative effort with clinician-investigators studying the Alagille syndrome, metaphase chromosomes and genomic DNA from affected individuals will be studied for subchromosomal deletions and for mutations in the candidate genes. Characterization of genes involved in Alagille syndrome could provide important insight into the pathophysiology of the disease, the development of normal liver and treatment of this disease. Recently, we and others found that mutations in Jagged1, a Notch1 receptor are responsible for Alagille Syndrome.


Condition
Alagille Syndrome

Genetics Home Reference related topics: Alagille syndrome
U.S. FDA Resources
Study Type: Observational
Official Title: Positional Cloning of the Gene(s) Responsible for Alagille Syndrome

Further study details as provided by National Institutes of Health Clinical Center (CC):

Estimated Enrollment: 225
Study Start Date: May 1997
Estimated Study Completion Date: March 2000
Detailed Description:

The goal of the project is to identify and clone the gene(s) responsible for the Alagille Syndrome (AGS) by a positional cloning approach. The first step towards this goal is to define the smallest genomic candidate region for AGS at 20p12 and to begin to identify genes within this region which are, by definition, candidate genes for the disease. In a collaborative effort with clinician-investigators studying the Alagille syndrome, metaphase chromosomes and genomic DNA from affected individuals will be studied for subchromosomal deletions and for mutations in the candidate genes. Characterization of genes involved in Alagille syndrome could provide important insight into the pathophysiology of the disease, the development of normal liver and treatment of this disease.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

All enrolled affected subjects, whose samples will be analyzed in this study, must meet the criteria for the clinical diagnosis of Alagille Syndrome (Syndromic Bile Duct Paucity) which include liver biopsy findings consistent with Alagille Syndrome and at least 3 of the 5 primary clinical criteria: cholestasis, characteristic face, posterior embryotoxon, "butterfly" vertebrae and cardiac findings.

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00001642

Locations
United States, Maryland
National Human Genome Research Institute (NHGRI)
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
  More Information

Publications:
Study ID Numbers: 970122, 97-HG-0122
Study First Received: April 6, 2000
Last Updated: March 3, 2008
ClinicalTrials.gov Identifier: NCT00001642  
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Chromosomal Deletion
Mutations
Paucity of Bile Ducts
Physical Map
Transcript Identification
Alagille Syndrome
Syndromic Bile Duct Paucity

Study placed in the following topic categories:
Chromosome Deletion
Liver Diseases
Cardiovascular Abnormalities
Alagille syndrome
Cholestasis
Cholestasis, Intrahepatic
Digestive System Diseases
Genetic Diseases, Inborn
Bile Duct Diseases
Alagille Syndrome
Biliary Tract Diseases
Abnormalities, Multiple
Congenital Abnormalities
Heart Defects, Congenital

Additional relevant MeSH terms:
Pathologic Processes
Disease
Syndrome
Cardiovascular Diseases

ClinicalTrials.gov processed this record on January 15, 2009