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

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

Requirements

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

Additional Information

Study Description at the National Institutes of Health
Biliary Atresia
Gastroenterology Research

Primary Investigator

Simon Horslen, MD

Contact Information

For more information about the study or enrollment, please contact:
Susan Richey, RN, Clinical Research Nurse Coordinator
412-692-6337