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97 : Phase 2 open‐label efficacy and safety study of the apical sodium‐dependent bile acid transporter inhibitor maralixibat in children with progressive familial intrahepatic cholestasis: 48‐week interim efficacy analysis

Researchers

Presenter

  • Richard J. Thompson

Principal Investigators

  • Deirdre A. Kelly

  • Patricia McClean

  • Alexander G. Miethke

  • Nisreen Soufi

  • Christine Rivet

  • Irena Jankowska

  • Cara L. Mack

  • Palaniswamy Karthikeyan

  • Joan Gu

  • Thomas Jaecklin

  • Robert H. Squires

  • Kathleen M. Loomes

Medical Centers

  • Institute of Liver Studies, Kings CollegeHospital NHS Trust, London , United Kingdom

  • Birmingham Children's Hospital, Birmingham, United Kingdom

  • Leeds General Infirmary, Leeds, United Kingdom

  • Division of Gastroenterology, Hepatology, and Nutrition, Cincinnati Childrens Hospital Medical Center, Cincinnati, OH

  • Department of Pediatric, University of Cincinnati School of Medicine, Cincinnati, OH

  • Pediatric Gastroenterology, Hepatology, and Nutrition, UCLA Medical Center, Los Angeles, CA

  • Department of Preventive Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA

  • Service d'Hépatologie, Gastroentérologie et Nutrition Pédiatriques, Hôpital Femme‐Mère‐Enfant, Bron, France

  • Gastroenterology, Hepatology, Eating Disorders and Pediatrics, Childrens Memorial Health Institute, Warsaw, Poland

  • Section of Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, University of Colorado School of Medicine

  • Shire Plc, Lexington, MA

  • Shire GmbH, Zug, Switzerland

  • Pediatric Gastroenterology, Hepatology and Nutrition, Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, PA

  • Department of Pathology, University of Pittsburgh, School of Medicine S- BST, Pittsburgh, PA, PA.

  • Division of Gastronterology, Hepatology, and Nutrition, Childrens Hospital of Philadelphia, Philadelphia, PA

  • Division of Hematology/Oncology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA

Locations

  • United Kingdom

  • United States

  • France

  • Poland

  • Switzerland

Companies

  • N/A

Study Components

Therapeutic Area

  • Gastroenterology (GU)

Disease

  • Familial cholestasis

  • Cirrhosis

Biomarkers

  • Alanine transaminase

Drug/Treatment

  • Lopixibat chloride

  • SHP625

Outcome

  • N/A

Study Design

  • N/A

Phase

  • II

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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