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Positive phase III trials for ileal bile acid transporter inhibitors

Nature Reviews Gastroenterology & Hepatology volume 21, page 462 (2024)Cite this article

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Two studies have reported positive phase III trials for ileal bile acid transporter inhibitors for pruritus and other cholestasis-associated clinical features. In the first study (ASSERT), the efficacy and safety of odevixibat was assessed for Alagille syndrome in a double-blind, randomized, placebo-controlled trial enrolling patients across ten countries. Patients (n = 52; median age 5.5 years) were randomly assigned to receive odevixibat (n = 35; 120 μg/kg per day) or placebo (n = 17) for 24 weeks. In the second study (MARCH-PFIC), a multicentre, randomized, double-blind, placebo-controlled study across 26 countries assessed the efficacy of maralixibat for progressive familial intrahepatic cholestasis (PFIC). Patients (n = 93; aged 1–17 years) were randomly assigned to take maralixibat (n = 47; 14 in the BSEP deficiency cohort, 33 in the all-PFIC cohort; starting dose 142.5 μg/kg, then escalated to 570 μg/kg) or a placebo (n = 46; 17 in the BSEP cohort, 31 in the all-PFIC cohort) twice-daily for 26 weeks. Both studies reported improvements in pruritus and reduced bile acid levels in the drug groups versus placebo. The most common adverse effect for both drugs was diarrhoea.

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  1. Nature Reviews Gastroenterology & Hepatology http://www.nature.com/nrgastro/

    Katrina Ray

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  1. Katrina Ray
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Correspondence to Katrina Ray.

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Ray, K. Positive phase III trials for ileal bile acid transporter inhibitors. Nat Rev Gastroenterol Hepatol 21, 462 (2024). https://doi.org/10.1038/s41575-024-00945-w

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