Biliary Atresia Study in Infants and Children (BASIC)
|First Received Date ICMJE||June 27, 2006|
|Last Updated Date||August 27, 2012|
|Start Date ICMJE||May 2006|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures ICMJE
||To identify the gene or genes implicated in the etiology of BA [ Time Frame: Specimens for this aim are collected once during study, usually at baseline. ] [ Designated as safety issue: No ]|
|Original Primary Outcome Measures ICMJE||Not Provided|
|Change History||Complete list of historical versions of study NCT00345553 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures ICMJE
|Original Secondary Outcome Measures ICMJE||Not Provided|
|Current Other Outcome Measures ICMJE||Not Provided|
|Original Other Outcome Measures ICMJE||Not Provided|
|Brief Title ICMJE||Biliary Atresia Study in Infants and Children|
|Official Title ICMJE||Biliary Atresia Study in Infants and Children (BASIC)|
Little is known about the factors that cause biliary atresia nor the factors that influence disease progression. The purpose of this study is to collect the pertinent clinical information, genetic material and body fluid samples to enable investigators to address the following aims: To identify the gene or genes implicated in the etiology of BA; To identify polymorphisms that may be important in disease progression such as HLA polymorphisms; To characterize the natural history of the older, non-transplanted child with BA.
Little is known about the factors that cause biliary atresia nor the factors that influence disease progression. A variety of genetic, autoimmune and environmental influences have been hypothesized to be important. Most studies to date have focused on the neonate and young child with BA, yet the older surviving child with BA can provide important information about genetics, as well as, natural history.
The purpose of this study is to collect the pertinent clinical information, genetic material and body fluid samples to enable investigators to address the following hypotheses:
Hypothesis 1: A genetic defect is a likely causative factor for BA among children with BA and multiple congenital anomalies.
Hypothesis 2: Autoimmune factors are likely to contribute to disease progression or acquisition and can be identified by correlating HLA among children with BA to healthy controls and by comparison of those who develop early complications including, variceal bleed, ascites, and growth failure compared to those who do not.
Hypothesis 3a: Sentinel events such as variceal bleeding, ascites and growth failure are earlier predictors of death or need for liver transplantation than the pediatric end-stage liver disease score (PELD) Hypothesis 3b: Health related quality of life will be impaired compared to healthy age matched children and relate to severity of illness.
Hypothesis 3c: Growth failure as measured by anthropometrics and nutritional supplementation will be predictive of onset of sentinel events (ascites, variceal bleed, death, and transplant) in the following 24 months.
This study will be performed by the Biliary Atresia Research Clinical Research Consortium (BARC), an NIDDK-funded network.
|Study Type ICMJE||Observational|
|Study Design ICMJE||Observational Model: Cohort
Time Perspective: Prospective
|Target Follow-Up Duration||Not Provided|
|Biospecimen||Retention: Samples With DNA
Samples of blood and urine will be collected for research purposes.
|Sampling Method||Non-Probability Sample|
The parents or guardians of all eligible subjects at each BARC center, or the subjects themselves if 18 years of age or older, will receive a letter of introduction, followed by a telephone call and, if willing, arrangement of an appointment at which time informed consent will be obtained. New patients who are at least one year of age and not participating in the BARC PROBE study will also be approached.
|Condition ICMJE||Biliary Atresia|
|Intervention ICMJE||Not Provided|
|Study Group/Cohort (s)||
|Publications *||Not Provided|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Recruiting|
|Estimated Enrollment ICMJE||1200|
|Estimated Completion Date||December 2012|
|Primary Completion Date||Not Provided|
|Eligibility Criteria ICMJE||
|Ages||1 Year to 75 Years|
|Accepts Healthy Volunteers||No|
|Location Countries ICMJE||United States, Canada|
|NCT Number ICMJE||NCT00345553|
|Other Study ID Numbers ICMJE||BASIC|
|Has Data Monitoring Committee||Yes|
|Responsible Party||National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|
|Study Sponsor ICMJE||National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|
|Collaborators ICMJE||Not Provided|
|Information Provided By||National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|
|Verification Date||August 2012|
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP