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An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
Sponsor: Mirum Pharmaceuticals, Inc.
Summary
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
Official title: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
Key Details
Gender
All
Age Range
1 Year - 18 Years
Study Type
INTERVENTIONAL
Enrollment
84
Start Date
2020-01-08
Completion Date
2025-04-23
Last Updated
2026-06-23
Healthy Volunteers
No
Interventions
Maralixibat
All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) twice daily
Locations (28)
Children's Hospital Los Angeles
Los Angeles, California, United States
Medstar Georgetown University Hospital
Washington D.C., District of Columbia, United States
Advent Health
Orlando, Florida, United States
Children's Hospital at Montefiore
The Bronx, New York, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
University of Texas, Health Science Center San Antonio
San Antonio, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Hospital Italiano de Buenos Aires
Buenos Aires, Argentina
Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde
Vienna, Austria
Cliniques Universitaires Saint-Luc
Brussels, Belgium
Sociedade Beneficente de Senhoras Hospital Sírio-Libanês
São Paulo, Brazil
University of Alberta - Women and Children's Health Research Institute
Edmonton, Alberta, Canada
Fundacion Cardioinfantil
Bogotá, Colombia
Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon
Lyon, France
CHU de Toulouse - Hôpital des Enfants
Toulouse, France
Medizinische Hochschule
Hanover, Germany
Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria
Bergamo, Italy
Ospedale Pediatrico bambino Gesu'
Roma, Italy
Hotel Dieu de France, Alfred Naccache
Beirut, Lebanon
Consultario de Joshue David Covarrubias Esquer
Zapopan, Mexico
Instytut Pomnik Centrum, Zdrowia Dziecka
Warsaw, Poland
KK Women's and Children's Hospital
Singapore, Singapore
Koc University Hospital
Istanbul, Turkey (Türkiye)
Birmingham Children's Hospital
Birmingham, United Kingdom
King's College Hospital NHS Foundation Trust
London, United Kingdom