Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)

PHASE2CompletedINTERVENTIONAL
Enrollment

31

Participants

Timeline

Start Date

October 28, 2014

Primary Completion Date

May 28, 2020

Study Completion Date

May 28, 2020

Conditions
Alagille Syndrome
Interventions
DRUG

LUM001 (Maralixibat)

LUM001, also known as Maralixibat (MRX) will be administered orally Once Daily (OD). To be administered Twice Daily (BID) for patients who are eligible.

DRUG

Placebo

Placebo will be administered orally once daily during randomized withdrawal period

Trial Locations (9)

261

Hospital Universitario La Paz- Hospital Materno Infantil, Madrid

2145

Children's Hospital Westmead, Westmead

3052

The Royal Children's Hospital Melbourne, Parkville

69677

Hopital Femme Mere Enfant De Lyon, Bron

75015

Hopital Necker-Enfants Malades, Paris

94275

Hopital Kremlin Bicetre, Paris

Unknown

Cliniques Universitaires Saint-Luc, Brussels

04-730

The Children's Memorial Health Institute, Warsaw

SE5 9RS

Paediatric Liver Center, Kings College Hospital, London

Sponsors
All Listed Sponsors
lead

Mirum Pharmaceuticals, Inc.

INDUSTRY