Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai

Mirum Pharmaceuticals, Inc.75 enrolled

Overview

A study to evaluate the efficacy and safety of maralixibat in infants with Biliary Atresia (BA) after Hepatoportoenterostomy (HPE, also known as the Kasai procedure).

This is a double-blind randomized, placebo-controlled study in subjects with Biliary Atresia with a primary endpoint at Week 26 followed by long-term open-label period during which all subjects will receive maralixibat to Week 104.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Biliary Atresia

Interventions

MaralixibatDRUG

A small molecule inhibitor of the ileal bile acid transporter (IBAT)

PlaceboOTHER

Identical to maralixibat except for the active drug substance

Eligibility

Sex: ALLMin age: 21 DaysMax age: 111 Days

Medical Language ↔ Plain English

Inclusion Criteria: 1. Male or female subjects with body weight ≥2500 g, who are ≥21 days old and \<90 days old at the time of HPE (Kasai) 2. HPE or Kasai Procedure within 3 weeks prior to randomization 3. Clinical diagnosis of biliary atresia Exclusion Criteria: 1. Subjects with intractable chronic diarrhea at randomization 2. Subjects not tolerating enteral feeds at randomization 3. History of ileal resection 4. Diagnosis of biliary atresia splenic malformation syndrome or cystic biliary atresia 5. Evidence of another non-biliary atresia pathology involving the intrahepatic bile duct (e.g., paucity, sclerosing cholangitis) 6. Evidence of liver failure (e.g. significant ascites)

Locations (22)

Phoenix Children's Division of Gastroenterology & Hepatology

Phoenix, Arizona, United States

Outcomes

Primary Outcomes

Mean Change in Total Serum Bilirubin Levels

Time frame: From baseline to Week 26

Secondary Outcomes

Mean Change in Total Serum Bile Acids

Time frame: From baseline to Week 26

Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26

Time frame: From baseline to Week 26

Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death.

Liver decompensation (hepatic encephalopathy, variceal bleeding, new persistent ascites)