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).

• Register: ClinicalTrials.gov
• Last refreshed on: 17 February 2025
• Main ID: NCT04729751
• Date of registration: 25/01/2021
• Prospective Registration: Yes
• Primary sponsor: Mirum Pharmaceuticals, Inc.
• Public title: 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). RISE
• Scientific title: 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
• Date of first enrolment: September 9, 2021
• Target sample size: 27
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT04729751
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

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

Key inclusion & exclusion criteria

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


Age minimum: 0 Days
Age maximum: 364 Days
Gender: All

Health Condition(s) or Problem(s) studied

Alagille Syndrome

Progressive Familial Intrahepatic Cholestasis

Cholestatic Liver Disease

Intervention(s)

Drug: Maralixibat

Primary Outcome(s)

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

Secondary Outcome(s)

To evaluate the effect on LSVs [Time Frame: From Baseline through to Week 13]

To evaluate the effect on liver enzymes (ALT, AST) and bilirubin [Time Frame: From Baseline through to Week 13]

Change in fasting serum bile acid (sBA) levels [Time Frame: From Baseline through to Week 13]

To assess the plasma level of maralixibat in infant participants [Time Frame: At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit]

Secondary ID(s)

2020-004628-40

MRX-801

Source(s) of Monetary Support

Please refer to primary and secondary sponsors