Gastroenterology Research

Researchers at UPMC Children’s Hospital of Pittsburgh have taken on multiple challenges in gastroenterology disorders. Pancreatitis, liver disease, biliary atresia, and inflammatory bowel disease are just a few of the many areas of focus.

With funding from the National Institutes of Health, Children’s gastroenterology laboratories provide leadership in: understanding the physiology of lipases – enzymes that digest fats – and, more specifically, the roles these proteins play in digestion and disease; characterizing the function and regulation of intestinal transporters for water and bile acids; and the pathophysiology and treatment of alpha-1-antitrypsin deficiency, the most common genetic disease of the liver in childhood.

Children’s gastroenterologists also head multiple clinical studies. Liver disease is under assault from Children’s pediatric gastroenterologists working to advance potential therapies that range from increasing our understanding of biliary atresia and acute liver failure, liver diseases often treated with liver transplantation, to transplanting hepatocytes as a possible cure for many childhood liver diseases. Clinical studies in pediatric inflammatory bowel disease are also a focus of Children’s gastroenterology researchers. These studies include participation in local and national registries to gather data about outcome and treatment of children with inflammatory bowel disease and an innovative and novel study to define the role of behavioral therapy in treating children with inflammatory bowel disease.

Children’s gastroenterology research is both local and international in scope, as the hospital has taken a leadership role in establishment of multicenter studies allowing collaboration and information sharing among experts throughout North America and beyond.


Clinical Studies

Alagille Syndrome & LUM001: The IMAGINE Study – Phase II

Long-term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTI), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects with Alagille Syndrome

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Alpha-1 Antitrypsin Deficiency Study – Phase II

A Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1-Antitrypsin Deficiency

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Biliary Atresia Long-Term Observation: The BASIC Study

Biliary Atresia Study in Infants, Children and Adults

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Cholestatic Liver Disease Treatment with Maralixibat over a Long Term: The MERGE Study – Phase II

A Long-Term Safety Study of Maralixibat, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor, in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study

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Cholestasis Long-term Observation: The LOGIC Study

Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)

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Cholestasis Natural History Database: The PROBE Study

A Prospective Database of Infants with Cholestasis

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Crohn’s Disease & its Effect on Growth in Boys & Girls

Sex Differences in Statural Growth Impairment in Pediatric Crohn’s Disease

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Crohn’s Disease & Sleep Disturbance Study – Phase IV

Treating Disrupted Sleep in Individuals with Inflammatory Bowel Disease: A Novel Adjunctive Therapy for Chronic Inflammatory Illness

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Crohn’s Disease Biomarker Study: The GEM Project

Study on the Genetic, Environmental and Microbial Interactions that Cause IBD (GEM Project)

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Crohn's Disease Treatment using Blood Stem Cells – Phase I/II

Autologous Stem Cell Transplantation with CD34 Selected Peripheral Blood Stem Cells (PBSC) in Pediatric and Young Adult Patients with Severe Crohn's Disease

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Hepatitis C Treatment with Elbasvir and Grazoprevir – Phase IIb

Study to Assess the Combination Regimen of Elbasvir/Grazoprevir in Pediatric Participants With Chronic Hepatitis C Infection

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Hepatitis C Treatment with Glecaprevir & Pibrentasvir – Phase II/III

An Open Label Multicenter Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Glecaprevir/Pibrentasvir in Pediatric Subjects with Genotypes 1-6 Chronic Hepatitis C Virus Infection

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IBD ImproveCareNow Registry

Using Patient Data to Transform Care and Improve Outcomes for Children, Adolescents and Young Adults with Inflammatory Bowel Disease

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ICARE Research Registry

Intestinal Care and Rehabilitation Center Database

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Liver Disease Scarring: The FORCE Study

FibroScan® in Pediatric Cholestatic Liver Disease Study

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Mitochondrial Hepatopathies Long-term Observation: The MITOHEP Study

Longitudinal Study of Mitochondrial Hepatopathies

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Nonalcoholic Fatty Liver/Steatohepatitis (NAFL/NASH) Observational Study

A 5-year Longitudinal Observational Study of Patients with Nonalcoholic Fatty Liver or Nonalcoholic Steatohepatitis

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Pancreatitis INSPPIRE 2 Study

Pediatric Longitudinal Cohort Study of Chronic Pancreatitis (INSPPIRE 2)

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Progressive Familial Intrahepatic Cholestasis Treatment with Maralixibat – Phase III

MRX-502: Randomized Double-blind Placebo-controlled Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis

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Progressive Familial Intrahepatic Cholestasis Treatment with Maralixibat over an Extended Term – Phase III

MRX-503: An Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis

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TPN-Related Cholestasis Treatment with Omegaven - Phase II

Compassionate Use of Omegaven in the Treatment of Parenteral Nutrition-Associated Hepatic Injury

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Additional Researchers

Arjumand Ghazi, PhD