Cholestatic Liver Disease Treatment with Maralixibat: The RISE Study – Phase II

Study to Evaluate Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome

Protocol Description

This multi-center international study will test maralixibat in infants with Alagille syndrome (ALGS) or progressive familial intrahepatic cholestasis (PFIC). Maralixibat has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of pruritus (itch) associated with ALGS in patients 1 year of age and older. This research studies the safety and tolerability of maralixibat, as well as its effects and side effects in children less than 12 months of age.

Study Level: Phase II

Eligibility Criteria

Subject to certain exclusion criteria, this study is open to infants diagnosed with ALGS or PFIC.
Boys and Girls: Newborns and babies less than 1 year old


For potential participants, the initial 4 weeks of the study will determine eligibility. Those accepted will receive the study drug once daily in a liquid oral form and attend regular clinic visits for 13 weeks. After that, participants may continue in the program for a longer-term extension period.
Visits: About 10 for the core study; Every 16 weeks for participants who remain in the long-term study after turning 1
Duration: 17 weeks to several years

Status: Open to Enrollment

Source(s) of Support

Mirum Pharmaceuticals

Primary Investigator

Simon Horslen, MD

Contact Information

For more information about the study or enrollment, please contact:
Laura Mathews, Clinical Research Coordinator
412-692-5811 or
Susan Richey, RN, Clinical Research Nurse Coordinator