A phase 3 clinical trial is currently recruiting participants with Alagille syndrome to test the safety and efficacy of odevixibat (Bylvay™).
Bylvay, manufactured by Albireo, is already approved by the US Food and Drug Administration (FDA) to treat pruritus in progressive familial intrahepatic cholestasis (PFIC).
This clinical trial, called ASSERT, is evaluating the treatment in up to 63 patients with Alagille syndrome of all ages.
Ron Cooper, the president and chief executive officer of Albireo, said in a business update, “A successful Bylvay launch is the main priority but we continue to advance and expand our portfolio with two additional phase 3 studies in biliary atresia and Alagille syndrome.” According to the company, the trial remains on track to report topline data in 2022.
During the double-blind, placebo-controlled study, participants will be randomized to receive either oral odevixibat or an identical-looking placebo once daily for 24 weeks.
The primary outcome measure of the trial is the change in scratching score from baseline. The secondary outcome measures are serum bile acid levels and safety and tolerability of the treatment as assessed by the number of participants with treatment-emergent adverse events and serious adverse events.
The trial is recruiting participants in the US, Canada, Belgium, France, the Netherlands, Poland, and the UK. It is expected to be completed in July 2022.
Odevixibat is a small molecule and selective ileal bile acid transport inhibitor that acts locally in the small intestine. Because there are fewer-than-normal bile ducts in Alagille syndrome, bile acids build up inside the liver and cause damage. It is thought that by reducing the reabsorption of bile acids from the small intestine to the liver, odevixibat could reduce the symptoms of Alagille syndrome, including pruritus. It could also delay or even prevent liver damage caused by the accumulation of bile acids.
Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT). ClinicalTrials.gov. December 19, 2020. Updated August 6, 2021. Accessed August 20, 2021.
Albireo reports Q2 financial results and business update. News release. Albireo; August 5, 2021.