90
Participants
Start Date
October 14, 2024
Primary Completion Date
October 31, 2026
Study Completion Date
February 28, 2027
Maralixibat
Maralixibat will be provided as an oral solution along with 0.5-, 1.0-, and 3.0-mL sized dosing dispensers. During the double-blind dose escalation period (4 weeks), the study drug (maralixibat) will be administered once daily for 1 week and then twice daily (BID; morning and evening). During the double-blind stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the double-blind dose-escalation period) of maralixibat. During the open-label dose escalation period (4 weeks), all participants will receive maralixibat treatment once daily for 1 week and then twice daily (BID; morning and evening). During the open-label stable dosing period (at least 16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the open-label dose-escalation period) of maralixibat.
Placebo
Placebo matched to maralixibat will be provided as an oral solution along with 0.5-, 1.0-, and 3.0-mL sized dosing dispensers. During the double-blind dose escalation period (4 weeks), study drug will be administered once daily for 1 week and then twice daily (BID; morning and evening). During the double-blind stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the double-blind dose-escalation period) of study drug. During the open-label dose escalation period (4 weeks), all participants will receive maralixibat treatment once daily for 1 week and then twice daily (BID; morning and evening). During the open-label stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the open-label dose-escalation period) of maralixibat.
RECRUITING
NYU Langone Health, New York
NOT_YET_RECRUITING
Mount Sinai Hospital, New York
RECRUITING
Morgan Stanley Children's Hospital - NewYork Presbyterian, New York
NOT_YET_RECRUITING
King Faisal Specialist Hospital & Research Center, Riyadh
RECRUITING
Hôpitaux Universitaires de Marseille Timone, Marseille
NOT_YET_RECRUITING
Children's Hospital of Philadelphia, Philadelphia
RECRUITING
Azienda Ospedaliera Papa Giovanni XXIII, Bergamo
NOT_YET_RECRUITING
Science 37, Inc (Remote-homebase Telemedicine), Morrisville
NOT_YET_RECRUITING
Vanderbilt University Medical Center, Nashville
NOT_YET_RECRUITING
Cincinnati Children's Hospital Medical Center, Cincinnati
RECRUITING
Lurie Children's Hospital, Chicago
RECRUITING
Ochsner Clinic Foundation, New Orleans
RECRUITING
Baylor College of Medicine, Houston
NOT_YET_RECRUITING
University of Utah, Salt Lake City
RECRUITING
Children's Hospital Los Angeles (CHLA), Los Angeles
RECRUITING
ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione, Palermo
RECRUITING
Stanford Children's Health in Palo Alto, Palo Alto
NOT_YET_RECRUITING
Seattle Children's Hospital, Seattle
RECRUITING
Hospital de Criança de Brasília (HCB), Brasília
RECRUITING
Hospital da Criança Santo Antonio, Porto Alegre
RECRUITING
Hospital Sírio-Libanês, São Paulo
RECRUITING
Hôpital Femme Mère Enfant, Lyon
RECRUITING
Hôpital Kremlin Bicêtre, Paris
NOT_YET_RECRUITING
Universitätsklinikum Hamburg Eppendorf - Klinik für Kinder- und Jugendmedizin, Hamburg
NOT_YET_RECRUITING
LMU Klinikum - Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Munich
RECRUITING
Hotel Dieu de France, Beirut
NOT_YET_RECRUITING
Hospital Infantil de México Federico Gómez, Mexico City
NOT_YET_RECRUITING
Consultario de Joshue David Covarrubias Esquer, Zapopan
RECRUITING
Instytut Pomnik Centrum Zdrowia Dziecka, Warsaw
RECRUITING
Hospital Sant Joan de Deu, Barcelona
RECRUITING
Hospital Universitario Vall d'Hebron, Barcelona
RECRUITING
Hospital Universitario La Paz, Madrid
RECRUITING
King's College Hospital NHS Foundation Trust, London
NOT_YET_RECRUITING
Stollery Children's Hospital, Edmonton
RECRUITING
Ospedale Pediatrico Bambino Gesu, Rome
Lead Sponsor
Mirum Pharmaceuticals, Inc.
INDUSTRY