Phase 2
N=75
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
Biliary Atresia
Bottom Line
View on ClinicalTrials.gov: NCT04524390 ↗Enrolled (actual)
75
Serious AEs
56.7%
Results posted
Feb 2025
Primary outcome: Primary: Mean Change in Total Serum Bilirubin Levels — -3.5; -3.11 mg/dl — p=0.7419
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Maralixibat (Drug); Placebo (Other)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Mirum Pharmaceuticals, Inc.
- Primary completion
- Nov 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Change in Total Serum Bilirubin Levels |
-3.5; -3.11 | 0.7419 |
| SECONDARY Mean Change in Total Serum Bile Acids |
-51.19; -5.29 | 0.2002 |
| SECONDARY Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26 |
24; 20 | 0.8412 |
| SECONDARY Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death. |
8; 7 | > 0.9999 |
| SECONDARY Proportion of Participants Undergoing Liver Transplantation or Death |
5; 3 | 0.6658 |
| SECONDARY Proportion of Participants Observed to Develop Clinically Evident Portal Hypertension Defined as Splenomegaly and Thrombocytopenia (Platelet Count <150 x 109/L) or Clinically Evident Ascites or Endoscopic Evidence of Esophageal or Gastric Varices. |
3; 4 | 0.6236 |
| SECONDARY Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL |
23; 18 | 0.6658 |
| SECONDARY Proportion of Participants With Mean sBA Levels ≤40 mmol/L |
10; 7 | 0.6659 |
Summary
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).
Eligibility Criteria
Inclusion Criteria
- Male or female subjects with body weight ≥2500 g, who are ≥21 days old and <90 days old at the time of HPE (Kasai)
- HPE or Kasai Procedure within 3 weeks prior to randomization
- Clinical diagnosis of biliary atresia
Exclusion Criteria
- Subjects with intractable chronic diarrhea at randomization
- Subjects not tolerating enteral feeds at randomization
- History of ileal resection
- Diagnosis of biliary atresia splenic malformation syndrome or cystic biliary atresia
- Evidence of another non-biliary atresia pathology involving the intrahepatic bile duct (e.g., paucity, sclerosing cholangitis)
- Evidence of liver failure (e.g. significant ascites)
Data sourced from ClinicalTrials.gov (NCT04524390). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.