A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
Completed · Phase 2
Conditions studied: Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Cholestatic Liver Disease
In brief
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 \[PFIC\].
Key facts
- Study ID
- NCT04729751
- Run by
- Mirum Pharmaceuticals, Inc.
- People needed
- 27
- Starts
- 2021-09-09
- Expected to finish
- 2024-12-17
- Last updated by the study team
- 2026-06-02
Who can join
Age: any, up to 1. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- Predicted complete absence of bile salt excretion pump (BSEP) function
- History of surgical disruption of the enterohepatic circulation
- History of liver transplant or imminent need for liver transplant
- Decompensated cirrhosis
- Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion
- Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study
Where it is running
- Children Hospital LA — Los Angeles, California, United States
- University of California - San Francisco — San Francisco, California, United States
- Medstar Georgetown University Hospital — Washington D.C., District of Columbia, United States
- Ochsner Hospital for Children — New Orleans, Louisiana, United States
- Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- Texas Children's Hospital — Houston, Texas, United States
- Seattle Children's Hospital — Seattle, Washington, United States
- Cliniques Universitaires Saint-Luc — Brussels, Belgium
- Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês — São Paulo, Brazil
- Hôpital Kremlin Bicêtre — Le Kremlin-Bicêtre, France
- Hopital Necker — Paris, France
- Consultorio de Joshue David Covarrubias Esquer — Zapopan, Mexico
- Instytut Pomnik-Centrum Zdrowia Dziecka — Warsaw, Poland
- King's College Hospital — London, United Kingdom
Full record on ClinicalTrials.gov
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