A new open-label, phase 2 study set to evaluate the safety and tolerability of maralixibat in infants with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis is currently recruiting infants under 12 months of age.

Following the US Food and Drug Administration (FDA) approval of maralixibat for the treatment of cholestatic pruritus in patients with ALGS aged 3 months or older, the initial study design enrolling patients older than 12 months has been updated.  

The researchers have also reduced the minimum age from 31 to 0 days for all participants outside of the US. Eligible patients will be those born with a gestational age of at least 36 weeks or a postmenstrual age of at least 36 weeks for those with gestational age between 32 and 36 weeks.

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Patients with a predicted complete absence of bile salt excretion pump function will be excluded from the study, as well as those with a history of surgical disruption of the enterohepatic circulation, liver transplant or imminent need for a liver transplant, decompensated cirrhosis or any other disease or condition that can interfere with the medication pharmacokinetics and metabolism, and any other relevant condition or abnormality.

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The current plan is to enroll at least 6 participants with ALGS or progressive familial intrahepatic cholestasis per each of the 2 cohorts. Maralixibat chloride in the form of an oral solution (ie, 5, 10, 15, or 20 mg/mL) will be self-administered twice per day for 13 weeks in the core study and for the duration of the long-term extension where applicable.

The researchers will monitor the frequency of treatment-emergent adverse events as the primary outcome measure. The updated secondary outcome measures, quantified from baseline to week 13, will evaluate the changes in fasting serum bile acid levels, liver enzymes (ALT and AST), bilirubin, and lipid-soluble vitamins, as well as assess the plasma level of maralixibat in infant participants at baseline, weeks 6, 10, and 13, or early at termination visit.

According to the most recent update, the study’s anticipated primary completion date is March 2023, and the actual study completion date has been extended from January to August 2023.


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). ClinicalTrials.gov. January 28, 2021. Updated March 29, 2023. Accessed March 29, 2023.