Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome

Who is this study for? Infants with cholestatic liver diseases including progressive familial intrahepatic cholestasis and Alagille syndrome
What treatments are being studied? Maralixibat
Status: Recruiting
Location: See all (14) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].

Eligibility
Participation Requirements
Sex: All
Maximum Age: 1
Healthy Volunteers: No
View:

• Body weight of ≥2.5 kg

• <12 months of age at the baseline visit (ROW). >31 days and <12 months of age at the baseline visit (US).

• Gestational age ≥36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required.

• Diagnosis of PFIC or ALGS

Locations
United States
California
Children Hospital LA
Recruiting
Los Angeles
University of California - San Francisco
Recruiting
San Francisco
Washington, D.c.
Medstar Georgetown University Hospital
Recruiting
Washington
Louisiana
Ochsner Hospital for Children
Recruiting
New Orleans
Pennsylvania
Children's Hospital of Pittsburgh
Recruiting
Pittsburgh
Texas
Texas Children's Hospital
Recruiting
Houston
Washington
Seattle Children's Hospital
Recruiting
Seattle
Other Locations
Belgium
Cliniques Universitaires Saint-Luc
Recruiting
Brussels
Brazil
Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês
Not yet recruiting
São Paulo
France
Hôpital Kremlin Bicêtre
Recruiting
Le Kremlin-bicêtre
Hopital Necker
Recruiting
Paris
Mexico
Consultorio de Joshue David Covarrubias Esquer
Not yet recruiting
Zapopan
Poland
Instytut Pomnik-Centrum Zdrowia Dziecka
Recruiting
Warsaw
United Kingdom
King's College Hospital
Recruiting
London
Contact Information
Primary
Clinical Trials Mirum
clinicaltrials@mirumpharma.com
+16506674085
Backup
Mirum Medical Information
medinfo@mirumpharma.com
Time Frame
Start Date: September 9, 2021
Estimated Completion Date: August 2023
Participants
Target number of participants: 12
Treatments
Experimental: Maralixibat
Participants will receive up to 600 μg/kg twice daily (PFIC) or up to 400 μg/kg once daily (ALGS) over 13 weeks in the core study and for the duration of the Long Term Extension (LTE) where applicable.
Sponsors
Leads: Mirum Pharmaceuticals, Inc.

This content was sourced from clinicaltrials.gov

Similar Clinical Trials