In patients with Alagille syndrome (ALGS), the key objectives are to evaluate the tolerability, long-term safety (including possible liver toxicity) and long-term efficacy of Livmarli treatment.
In patients with Progressive Familial Intrahepatic Cholestasis (PFIC), the key objectives are to evaluate tolerability, long-term safety (including possible liver toxicity and potential impact of chronic exposure to propylene glycol (PG)), long-term efficacy, and growth and development.
Understand and execute an Informed consent and assent (as applicable)
For participants with ALGS ≥2 months of age at Day 1
For participants with PFIC, ≥3 months of age at Day 1
A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis, or a clinically and/or genetically confirmed PFIC diagnosis
History of Liver Transplant
Any Livmarli contraindications (as per SmPC)
Any condition or abnormality that, in the opinion of the investigator, may interfere with the participation in or completion of the study
Received an investigational drug within 30 days before the first dose of Livmarli (Participation in previous maralixibat studies or expanded-access programs is acceptable.)
Single group
Livmarli Oral Product
DrugParticipants will be treated according to standard of care for the duration of the study and Schedule of Assessments.
Adverse Events (AEs)
Number and proportion of participants with AEs.
Liver function tests (LFTs) - measuring enzyme activity or concentration
Change in LFTs from Baseline: Total and direct bilirubin
Liver function tests (LFTs) - measuring enzyme activity or concentration
Change in LFTs from Baseline: Aminotransferases and GGT.
Fat-Soluble Vitamins (FSV) Concentration
Change in FSV levels (vitamins A, D, and E) from Baseline.
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