Phase 3 ASSERT study topline results and phase 3 ASSERT extension study interim results demonstrate rapid and sustained improvements in sleep disturbance, pruritus, and bile acids concentration in patients with Alagille syndrome (ALGS) treated with odevixibat.
Results from both studies were presented at the 55th Annual Meeting of the European Society for Paediatric Gastroenterology, Hepatology, and Nutrition (ESPGHAN 2023) that took place in May in Vienna.
In the ASSERT study, 52 patients with ALGS with a history of significant pruritus and elevated bile acids were randomly assigned to groups receiving either 120 μg/kg per of odevixibat or placebo. The researchers looked for changes in observer-reported scratching score, sleep parameters, and serum bile acids levels. They also observed the occurrence of treatment-emergent adverse events.
In the extension study, patients who completed the ASSERT study received 120 μg/kg of odevixibat daily. Patients who had previously received the experimental drug and treatment-naive patients who had previously taken a placebo were monitored for pruritus, serum bile acids, sleep, and treatment-emergent adverse events.
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According to the study results, patients treated with odevixibat experienced statistically significant and clinically meaningful improvements in pruritus and multiple sleep parameters, and their serum concentration of bile acids lessened significantly. The incidence of adverse events was similar in both groups, whereas those receiving the study drug reported diarrhea more often.
The results of the ASSERT-EXT study were consistent with the findings from the previous study. Treatment-naive patients experienced rapid improvements in pruritus, sleep, and bile acids, and those who had previously received odevixibat showed sustained improvements. In both studies, the medication was well-tolerated.
“ALGS is a rare, multisystem disorder caused by mutations in JAG1 or NOTCH2 with hepatic manifestations that include elevated bile acids and pruritus associated with impaired sleep and quality of life,” the study authors explained.
Ovchinsky N. Efficacy and safety of odevixibat in patients with Alagille syndrome: top-line results from ASSERT, a phase 3, double-blind, randomized, placebo-controlled study. Presented at: 55th Annual Meeting of the European Society for Paediatric Gastroenterology, Hepatology, and Nutrition (ESPGHAN), Vienna, Austria; May 18, 2023.
Ovchinsky N. Efficacy and safety of odevixibat in patients with Alagille syndrome: interim results from the open-label, phase 3 ASSERT-EXT study. Presented at: 55th Annual Meeting of the European Society for Paediatric Gastroenterology, Hepatology, and Nutrition (ESPGHAN), Vienna, Austria; May 19, 2023.