ASSERT Trial: Odevixibat in Alagille Syndrome

assert trial odevixibat

ASSERT Trial Highlights Efficacy of Odevixibat in Alagille Syndrome

In a significant stride towards better management of Alagille Syndrome, the ASSERT trial has recently showcased promising results for the ileal bile acid transporter inhibitor, Odevixibat. This phase 3, double-blind, randomized, placebo-controlled trial aimed to evaluate the efficacy and safety of Odevixibat in patients suffering from Alagille syndrome, a genetic disorder that can affect the liver and other systems of the body.

Study Design

The ASSERT trial enrolled 52 patients diagnosed with Alagille syndrome who were under 18 years of age. These participants were selected based on a history of significant pruritus (itchiness) and elevated serum bile acid concentration, among other criteria. They were randomized to receive either Odevixibat (120 µg/kg per day) or a placebo.


The primary outcome measured was the change in caregiver-reported scratching score from baseline to weeks 21-24. The results were significantly in favor of Odevixibat, which showed a decrease of -1.7 in the scratching score compared to -0.8 in the placebo group. This translated to a statistically significant difference with a P-value of 0.0024.

In terms of secondary outcomes, Odevixibat led to a significant reduction in serum bile acid concentrations. From baseline to an average of weeks 20 and 24, patients on Odevixibat experienced a reduction of -90 µmol/L compared to a reduction of -22 µmol/L in the placebo group, with a P-value of 0.0012.


The ASSERT trial concludes that Odevixibat could serve as an efficacious non-surgical intervention to improve pruritus, reduce serum bile acids, and potentially enhance the standard of care in patients with Alagille syndrome. These findings could herald a new era of treatment for this challenging genetic disorder, providing relief and improved quality of life for affected patients and their families.

This pivotal trial paves the way for further research into the long-term benefits and safety of Odevixibat, setting a new benchmark in the therapeutic landscape of Alagille syndrome.

For more detailed information, the full results of the ASSERT trial can be accessed through the Lancet publication.

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