A Phase 4 interventional study of Livmarli Oral Product in Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis, sponsored by Mirum Pharmaceuticals, Inc.. Recruiting at 15 sites in 8 countries. Open to participants aged 2 Months and older. Per ClinicalTrials.gov, last updated 2026-06-23.
Sponsored by Mirum Pharmaceuticals, Inc. · Phase 4, Interventional, and Other
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.
This is a multicenter, open-label, low-intervention clinical study in participants diagnosed with either ALGS with cholestatic pruritus or PFIC who are treated with Livmarli.
Participants will be treated according to standard of care for the duration of the study.
31 studies on the registry are indexed under Alagille Syndrome; 11 are open to participants now.
This study's planned enrollment of 230 is above the median of 31 across 15 interventional studies indexed under Alagille Syndrome.
Browse Alagille Syndrome studies →Mirum Pharmaceuticals, Inc. is the lead sponsor of 40 studies on the registry; 7 are open to participants now.
Of its 12 completed or terminated interventional studies of FDA-regulated products, 11 (92%) have results posted.
Counted across the registry records on this site, refreshed daily.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALGS: Primary cohort with a minimum of 45 previously untreated (treatment naïve). Supplemental cohort of participants who have previously received Livmarli. PFIC: Patients with Progressive Familial Intrahepatic Cholestasis treated with Livmarli. The study will enroll a minimum of 130 patients with PFIC
Drug: Livmarli Oral Product
Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.
Also known as: Maralixibat
Adverse Events (AEs)
Number and proportion of participants with AEs.
Time frame: Up to 7 days after the last dose of Livmarli.
Liver function tests (LFTs) - measuring enzyme activity or concentration
Change in LFTs from Baseline: Total and direct bilirubin
Time frame: Once every 3-4 months, for 5 years.
Liver function tests (LFTs) - measuring enzyme activity or concentration
Change in LFTs from Baseline: Aminotransferases and GGT.
Time frame: Once every 3-4 months, for 5 years.
Fat-Soluble Vitamins (FSV) Concentration
Change in FSV levels (vitamins A, D, and E) from Baseline.
Time frame: Once every 3-4 months, for 5 years.
International Normalized Ratio (INR)
Change in INR level from Baseline.
Time frame: Once every 3-4 months, for 5 years.
Long-Term Clinical Outcomes
Number of participants with surgical biliary diversion, liver transplantation, liver transplant waitlist status change, clinically evident portal hypertension, complications of liver cirrhosis, liver carcinoma, liver decompensation, and death.
Time frame: Once every 3-4 months, for 5 years.
Laboratory Test for Monitoring of Propylene Glycol Toxicity in Participants with PFIC
Change in PG level from Baseline.
Time frame: Once every 3-4 months, for 5 years.
Started Sep 2025 (actual). Primary completion is estimated for Sep 2030, 59 months after the start.
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Studies of the drug tested here: Maralixibat