Biliary Atresia Treatment with Maralixibat after Kasai Procedure: The EMBARK Study – Phase II

Study to Evaluate Maralixibat in the Treatment of Subjects with Biliary Atresia after Hepatoportoenterostomy

Protocol Description

This multi-center international study will investigate whether the drug maralixibat can improve liver health and delay or reduce the need for a future liver transplant for babies who recently had a hepatoportoenterostomy, also called the Kasai procedure, for biliary atresia. In this rare disease, the ducts that drain bile from the liver are blocked, resulting in a build-up of bile that can cause liver damage. The Kasai procedure bypasses blocked ducts helping the liver to work better, but only delaying the need for a liver transplant. Maralixibat reduces the level of bile in the body, which may help reduce liver damage caused by too much bile.

Study Level: Phase II

Eligibility Criteria

This study is open to babies diagnosed with biliary atresia who recently had an operation called the Kasai procedure.
Boys and Girls: Babies ages 21 to 111 days


Half the study participants will receive maralixibat daily and the other half will receive a placebo for 6 months. After that, all study participants will receive maralixibat for the remainder of the study. After 2 years, children may be able to continue in the program, unless maralixibat becomes available by prescription, the study is stopped, or the child’s doctor recommends discontinuing participation.
Visits: About 22, many of which will be phone calls rather than in-person visits if specific lab work or other physical examinations are not required
Duration: About 2 years

Status: Enrolling by Invitation

Source(s) of Support

Mirum Pharmaceuticals

Primary Investigator

Simon Horslen, MD

Contact Information

For more information about the study or enrollment, please contact:
Susan Richey, RN, Clinical Research Nurse Coordinator