# BCM/TCH CHOLESTATIC LIVER DISEASE CONSORTIUM

> **NIH NIH U01** · BAYLOR COLLEGE OF MEDICINE · 2021 · $396,714

## Abstract

Project Summary
Cholestatic liver diseases including Alagille syndrome, alpha-1 antitrypsin deficiency, bile acid synthesis
defects, biliary atresia, cystic fibrosis, mitochondrial hepatopathies, progressive familial intrahepatic cholestasis
and primary sclerosing cholangitis, lead to significant morbidity and mortality in childhood, frequently
necessitating liver transplantation. No single North American clinical center sees a large enough number of
patients with these disorders to permit a rigorous approach to addressing unresolved questions including
etiology and pathogenesis, optimal methods of diagnosis and treatment, and factors that influence disease
severity and prognosis. This competitive renewal proposal from the Baylor College of Medicine and Texas
Children's Hospital (BCM/TCH) seeks to continue ongoing research activities in the Childhood Liver Disease
Research Network (ChiLDReN). This application for renewal funding includes a strong commitment to
continuing the on-going research efforts and two new proposals, a novel pilot and feasibility clinical trial applied
to biliary atresia and a translational protocol focused on the pathogenesis of sclerosing cholangitis. The clinical
center at BCM/TCH includes an outstanding group of clinician investigators with an extensive track record in
synergistic translational and clinical research relevant to pediatric liver diseases. Performance to date in the
on-going studies of ChiLDReN has been exemplary and has taken full advantage of a predominant market
share of the population base of the Houston metropolitan region (5th largest in the United States) and the large
referral patterns to TCH as a quaternary center for Pediatric Hepatology and Liver Transplantation. A pilot and
feasibility trial of intravenous NAC will be conducted as an open label investigation in infants undergoing
hepatoportoenterstomy for biliary atresia (IND 135796 and NCT03499249). The primary outcome measure will
focus on biomarkers of bile flow. Promising findings in this pilot study could be expanded to a larger scale
multi-center investigation as part of ChiLDReN. The proposed translational protocol will use metagenomic
whole genome sequencing to assess the bacterial fecal microbiome in children with sclerosing cholangitis and
correlate microbial signatures and bacterial gene expression patterns with clinical phenotypes characterized in
the newly developed prospective observational study of primary sclerosing cholangitis in children.

## Key facts

- **NIH application ID:** 10200025
- **Project number:** 5U01DK103149-08
- **Recipient organization:** BAYLOR COLLEGE OF MEDICINE
- **Principal Investigator:** BENJAMIN L SHNEIDER
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $396,714
- **Award type:** 5
- **Project period:** 2014-09-10 → 2024-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10200025

## Citation

> US National Institutes of Health, RePORTER application 10200025, BCM/TCH CHOLESTATIC LIVER DISEASE CONSORTIUM (5U01DK103149-08). Retrieved via AI Analytics 2026-05-21 from https://api.ai-analytics.org/grant/nih/10200025. Licensed CC0.

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