EMBARK Evaluation of Maralixibat BiliaryAtresia Response post Kasai
Research type
Research Study
Full title
Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Biliary Atresia after Hepatoportoenterostomy
IRAS ID
287291
Contact name
Lara Longpre
Contact email
Sponsor organisation
Mirum Pharmaceutical Inc.
Eudract number
2020-000974-22
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 1 months, 16 days
Research summary
The EMBARK Study is a 2-phase trial to assess the safety and effectiveness of maralixibat in infants with biliary atresia who have recently undergone Hepatoportoenterostomy (HPE) surgery, also known as the Kasai procedure.
Biliary atresia is a rare liver disease affecting infants in which a blockage occurs in the ducts that carry bile from the liver to the gallbladder. It presents shortly after birth and can lead to various complications related to bile flow and liver damage. Biliary atresia is the leading cause of liver transplant in children worldwide.
To be eligible for the study, patients must be aged at least 21 days but less than 90 days old at the time of HPE/Kasai surgery. Surgery must be performed within 3 weeks of the start of study treatment and patients must be at least 31 days old at this time.
The first phase of the EMBARK Study will be a double-blind period, where the participants will receive either maralixibat or placebo for a period of 26 weeks. Participants will then proceed to the open-label extension (OLE) period of the study, where all patients will receive maralixibat. Participants will continue in the OLE until they become eligible for a long term maralixibat study, the drug is commercially available, the sponsor stops the program or the study doctor decides to the discontinue the child from the trial.
Maralixibat is a grape flavoured oral solution that will be administered to the child twice a day. More than 1600 individuals have received maralixibat, including more than 120 children with other types of liver disease. Maralixibat may be able to help to reduce the amount of bile acids in a child’s blood, helping prevent liver damage and the need for future liver transplant.
This trial will be conducted in NHS sites within Paediatric departments.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
20/SC/0400
Date of REC Opinion
23 Apr 2021
REC opinion
Further Information Favourable Opinion