A phase 3 pivotal clinical trial testing the safety and efficacy of odevixibat (Bylvay™) in patients with Alagille syndrome, called ASSERT, has completed enrolling participants, announced Albireo, the developer of the treatment. 

“Families coping with Alagille syndrome are deeply in need of more therapeutic options, as evidenced by the completed enrollment for our phase 3 ASSERT study, consistent with guidance, while exceeding our original enrollment target,” said Ron Cooper, Albireo’s president and chief executive officer in a press release from the company.

The company also announced it is opening an expanded access program for patients with Alagille syndrome to receive odevixibat.


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ASSERT is a double-blind, randomized, placebo-controlled study investigating the safety and efficacy of odevixibat compared to placebo. Participants will receive oral capsules once a day for 24 weeks.

The primary outcome measure is the change from baseline in scratching score. Secondary outcome measures are serum bile acid levels and safety and tolerability as assessed by the number of participants with treatment-emergent adverse events or serious adverse events.

Topline results from the study are expected by the end of 2022. 

Read more about odevixibat

Alagille syndrome is characterized by intrahepatic bile duct paucity causing bile acid accumulation in the liver, which leads to chronic cholestasis and pruritus.  

Odevixibat is a nonsystemic ileal bile acid transport inhibitor. It works by decreasing the reabsorption of bile acids from the small intestine to the liver thereby reducing symptoms of such as pruritus. It is hoped that the treatment may also delay or prevent liver damage caused by toxic levels of bile acid accumulation.

The treatment is already approved in the US to treat pruritus in patients with progressive familial intrahepatic cholestasis.

References

Albireo completes enrollment in pivotal phase 3 ASSERT study of BylvayTM (odevixibat) in Alagille syndrome. News release. Albireo; March 29, 2022.

Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT). US National Library of Medicine. Last updated February 21, 2022. Accessed March 31, 2022.