A new clinical trial has started recruiting participants to evaluate the safety and tolerability of maralixibat (Livmarli™) in infants with cholestatic liver diseases including Alagille syndrome.

The phase 2 open-label study called RISE is sponsored by Mirum Pharmaceuticals and aims to assess whether maralixibat is safe and well-tolerated in children aged less than 1 year who have Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC).

Patients up to 364 days old weighing at least 2.5 kg with a diagnosis of Alagille syndrome or PFIC are eligible to participate at locations in the US, Belgium, France, Poland, and the UK. 


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Participants with Alagille syndrome will receive up to 400 μg/kg of maralixibat once a day over 13 weeks in the core portion of the study and for the duration of the long-term extension where applicable. 

The primary outcome measure will be the frequency of treatment-emergent adverse events. The secondary outcome measure will be the change in fasting serum bile acid levels from baseline through week 13.

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The estimated primary completion date of the study is June 2022. The estimated study completion date is January 2023. 

Maralixibat is an oral solution that inhibits the action of apical sodium-dependent bile acid transporter (ASBT), a protein that plays a key role in the enterohepatic circulation of bile acids. It is approved by the US Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients with Alagille syndrome aged 1 year and older.

Alagille syndrome is characterized by intrahepatic bile duct paucity leading to the buildup of bile in the liver and liver damage. 

The inhibition of ASBT by maralixibat results in more bile acids being excreted in the feces and therefore reducing the levels of systemic bile acids.

Reference

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). US National Library of Medicine. Last updated October 20, 2021. Accessed November 23, 2021.