Phase 2
N=27
A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
Progressive Familial Intrahepatic Cholestasis · Alagille Syndrome · Cholestatic Liver Disease
Bottom Line
View on ClinicalTrials.gov: NCT04729751 ↗Enrolled (actual)
27
Serious AEs
29.6%
Results posted
Jun 2026
Primary outcome: Primary: Frequency of Treatment-emergent Adverse Events [TEAEs] — 16; 10; 4; 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Maralixibat (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Mirum Pharmaceuticals, Inc.
- Primary completion
- Dec 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Frequency of Treatment-emergent Adverse Events [TEAEs] |
16; 10; 4; 3; 6; 1 | — |
| SECONDARY Change in Fasting Serum Bile Acid (sBA) Levels |
292.81; 228.32; 172.49; 138.90; -75.68; -77.49 | — |
| SECONDARY To Evaluate the Effect on Liver Enzymes (ALT) |
167.35; 149.00; 256.87; 80.50; 99.00; -27.00 | — |
| SECONDARY Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin E |
6.54; 2.78; 5.99; 3.98; -0.46; 0.92 | — |
| SECONDARY Maximum Level of Maralixibat Concentration in Plasma From Baseline to Week 13 for ALGS |
0.5576 | — |
| SECONDARY To Evaluate the Effect on Liver Enzymes (AST) |
184.12; 159.10; 236.8; 85.71; 69.27; -30.43 | — |
| SECONDARY To Evaluate the Effect on Bilirubin |
142.54; 41.02; 114.89; 18.51; -22.85; -18.01 | — |
| SECONDARY Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin A |
1.78; 1.50; 2.08; 2.32; 0.45; 0.61 | — |
| SECONDARY Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamins D and K |
62.64; 62.87; 78.42; 94.84; 7.63; 20.65 | — |
| SECONDARY Maximum Level of Maralixibat Concentration in Plasma From Baseline to Week 13 for PFIC |
0.153 | — |
Summary
This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].
Eligibility Criteria
Inclusion Criteria
- Body weight of ≥2.5 kg
- 31 days and <12 months of age at the baseline visit (US).
- Gestational age ≥36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required.
- Diagnosis of PFIC or ALGS
Exclusion criteria
- Predicted complete absence of bile salt excretion pump (BSEP) function
- History of surgical disruption of the enterohepatic circulation
- History of liver transplant or imminent need for liver transplant
- Decompensated cirrhosis
- Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion
- Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study
Data sourced from ClinicalTrials.gov (NCT04729751). 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.