An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

PHASE2CompletedINTERVENTIONAL
Enrollment

34

Participants

Timeline

Start Date

March 16, 2015

Primary Completion Date

June 1, 2020

Study Completion Date

June 1, 2020

Conditions
Alagille Syndrome
Interventions
DRUG

LUM001 (Maralixibat)

Dosing of LUM001 also known as Maralixibat (MRX) with the objective of achieving optimal control of pruritus at a dose level that is tolerated by the participant and up to a maximum daily dose of 280 micrograms per kilogram (mcg/kg).

Trial Locations (11)

15224

Children's Hospital of Pittsburgh of UPMC, Pittsburgh

19147

The Children's Hospital of Philadelphia, Philadelphia

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

46202

Riley Hospital for Children, Indianapolis

77030

Baylor College of Medicine/Texas Children's Hospital, Houston

80045

Children's Hospital Colorado, Aurora

84113

University of Utah, Salt Lake City

90027

Children's Hospital Los Angeles, Los Angeles

94143

University of California at San Francisco Children's Hospital, San Francisco

98105

Seattle Children's Hospital, Seattle

M5G 1X8

The Hospital for Sick Children, Toronto

Sponsors
All Listed Sponsors
collaborator

Lumena Pharmaceuticals, Inc.

INDUSTRY

collaborator

Childhood Liver Disease Research and Education Network

OTHER

lead

Mirum Pharmaceuticals, Inc.

INDUSTRY