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).

Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04729751

Acronym

RISE

Enrollment

Registered

2021-01-28

Start date

2021-09-09

Completion date

2024-12-17

Last updated

2025-02-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Cholestatic Liver Disease

Keywords

PFIC, ALGS, Maralixibat, Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases, Pediatric

Brief 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 \[PFIC\].

Detailed description

This is an open label study where all participants will receive maralixibat treatment.

Interventions

DRUGMaralixibat

Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL) * 400 μg/kg maralixibat chloride is equivalent to 380 µg/kg maralixibat free base * 600 μg/kg maralixibat chloride is equivalent to 570 µg/kg maralixibat free base

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Intervention model description

Single group with 2 cohorts - ≥ 6 participants each from ALGS and PFIC.

Eligibility

Sex/Gender

ALL

Age

0 Days to 364 Days

Healthy volunteers

Inclusion criteria

1. Body weight of ≥2.5 kg 2. \<12 months of age at the baseline visit (ROW). \>31 days and \<12 months of age at the baseline visit (US). 3. 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. 4. Diagnosis of PFIC or ALGS

Exclusion criteria

1. Predicted complete absence of bile salt excretion pump (BSEP) function 2. History of surgical disruption of the enterohepatic circulation 3. History of liver transplant or imminent need for liver transplant 4. Decompensated cirrhosis 5. 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 6. 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

Design outcomes

Primary

Measure	Time frame
Frequency of treatment-emergent adverse events [TEAEs]	From Baseline through to Week 13

Secondary

Measure	Time frame
Change in fasting serum bile acid (sBA) levels	From Baseline through to Week 13
To evaluate the effect on liver enzymes (ALT, AST) and bilirubin	From Baseline through to Week 13
To evaluate the effect on LSVs	From Baseline through to Week 13
To assess the plasma level of maralixibat in infant participants	At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit

Countries

Belgium, Brazil, France, Mexico, Poland, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026