A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)

PHASE3CompletedINTERVENTIONAL
Enrollment

93

Participants

Timeline

Start Date

July 9, 2019

Primary Completion Date

September 1, 2022

Study Completion Date

September 1, 2022

Conditions
Progressive Familial Intrahepatic Cholestasis (PFIC)
Interventions
DRUG

Maralixibat

Maralixibat oral solution (up to 600 mcg/kg) orally twice daily for 26 weeks.

OTHER

Placebo

Placebo matching to maralixibat orally twice daily for 26 weeks.

Trial Locations (31)

1083

Semmelweis Egyetem - Altalanos Orvostudomanyi Kar (SE AOK), Budapest

10461

The Children's Hospital at Montefiore Yeshiva University - Montefiore Medical Center, New York

15224

UPMC Children's Hospital of Pittsburgh, Pittsburgh

29425

Medical University of South Carolina, Charleston

31059

CHU de Toulouse - Hôpital des Enfants, Toulouse

32803

Advent Health, Orlando

44195

Cleveland Clinic - Pediatric Institute, Cleveland

45229

Cincinnati Children's Hospital, Cincinnati

75390

University of Texas - UT Southwestern Medical Center, Dallas

78229

University of Texas, Health Science Center San Antonio, San Antonio

90027

Children's Hospital Los Angeles CHLA, Los Angeles

98105

Seattle Children's Hospital, Seattle

20007-2113

Medstar Georgetown University Hospital, Washington D.C.

Unknown

Hospital Italiano de Buenos Aires, Buenos Aires

Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde, Vienna

Universite Catholique de Louvain (UCLouvain) - Cliniques Universitaires Saint-Luc, Brussels

Sociedade Beneficente de Senhoras Hospital Sírio-Libanês, São Paulo

University of Alberta - Women and Children's Health Research Institute, Edmonton

Fundacion Cardioinfantil - Departamento de Investigaciones, Bogotá

Hospices Civils de Lyon - Hopital Femme Mere Enfant Service de Gastroenterologie, Hepatologie et Nutrition, Lyon

CHU de Marseille, Hôpital de la Timone, Marseille

Medizinische Hochschule Hannover, Hanover

Hospital Papa Giovanni XXIII / Unità di Pediatria, Bergamo

Ospedale Pediatrico Bambino Gesu, Roma

Hotel Dieu de France, Beirut

Consultario de Joshue David Covarrubias Esquer, Zapopan

Instytut Pomnik Centrum Zdrowia Dziecka, Warsaw

KK Women's and Children's Hospital, Singapore

Koc University Hospital, Istanbul

Birmingham Children's Hospital, Birmingham

King's College Hospital NHS, London

Sponsors
All Listed Sponsors
lead

Mirum Pharmaceuticals, Inc.

INDUSTRY