Biliary Atresia Study in Infants and Children

Официальное название

Biliary Atresia Study in Infants and Children (BASIC)

Обзор

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 characterize the natural history of the older, non-transplanted child with BA.

Подробное описание

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 2a: 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 2b: Health related quality of life will be impaired compared to healthy age matched children and relate to severity of illness.

Hypothesis 2c: 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 Childhood Liver Disease Research Network (ChiLDReN), a National Institute of Diabetes \& Digestive and Kidney Diseases (NIDDK) funded network.

Первичные конечные точки

• To identify the gene or genes implicated in the etiology of BA [Срок оценки: Specimens for this aim are collected once during study, usually at baseline.]

Вторичные конечные точки (1)

• Define the natural history of the older, non-transplanted child with biliary atresia [Срок оценки: Observational information collected at entrance into study as well as at each yearly follow-up visit.]

Критерии участия

Критерии включения

• Participants need to have a confirmed diagnosis of BA determined by chart review including review of pertinent diagnostic biopsy reports, radiologic reports and surgical reports (if surgery was performed).
• Participants need to be >6 months of age up to and equal to the age of 20 (participants enrolled at 20 years of age will have one visit).
• Participants with their native liver.
• Parent, guardian or participant (if 18 years of age or older) is willing to provide informed consent and, when appropriate, the participant is willing to assent.

Критерии исключения

• Currently participating in the ChiLDReN study PROBE.
• Inability to confirm original diagnostic evaluation of biliary atresia.
• Inability or unwillingness of family or participant to participate in all scheduled visits.
• History of liver transplantation.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 15 центров

• Children's Hospital of Los Angeles — Los Angeles
• University of California at San Francisco — San Francisco
• Children's Hospital Colorado — Aurora
• Children's Healthcare of Atlanta - Emory University — Atlanta
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
• Riley Children's Hospital — Indianapolis
• Johns Hopkins School of Medicine — Baltimore
• Washington University School of Medicine — St Louis
• … и ещё 7 центров

Канада · 1 центр

• Hospital for Sick Children — Toronto

Публикации

• Ng VL, Haber BH, Magee JC, Miethke A, Murray KF, Michail S, Karpen SJ, Kerkar N, Molleston JP, Romero R, Rosenthal P, Schwarz KB, Shneider BL, Turmelle YP, Alonso EM, Sherker AH, Sokol RJ; Childhood Liver Disease Research and Education Network (CHiLDREN). Medical status of 219 children with biliary atresia surviving long-term with their native livers: results from a North American multicenter cons PMID 25015575

Идентификаторы

NCT: NCT00345553 · BASIC Study - ChiLDReN Network · U01DK103149 · U01DK103140 · U01DK103135 · U01DK084575 · U01DK084538 · U01DK084536 · U01DK062503 · U01DK062500 · U01DK062497 · U01DK062481 · U01DK062470 · U01DK062466 · U01DK062456 · U01DK062453 · U01DK062452 · U01DK062445 · U01DK062436 · U24DK062456

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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