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

MRX-701

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).

2021-07-08

2023-11-07

2024-02-07

Completed

Early phase I

75

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)

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}}, 'enrollmentInfo': {'count': 75, 'type': 'ACTUAL'}}

2024-03-08