A new open-label phase 3 clinical trial evaluating the long-term safety and efficacy of odevixibat (A4250) in patients with Alagille syndrome (ALGS) is currently recruiting participants by invitation in the US, France, Poland, and the UK.

The trial called ASSERT-EXT is enrolling an estimated 63 adults and children with the disease who completed the 24-week treatment period of study A4250-012, a phase 3 double-blind, randomized, placebo-controlled study of the safety and efficacy of the treatment. Participants will receive oral odevixibat once a day for 72 weeks.

The primary outcome measure is the change in pruritus from baseline to week 72. Secondary outcome measures include the change in serum bile acids, quality of life, sleep parameters, and global symptom relief as well as the safety and tolerability of the disease from baseline to week 72.

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Patients with decompensated liver disease, a history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy are not eligible. Full eligibility criteria can be found on the clinical trial’s website. The study is estimated to be completed in October 2023.

Read more about odevixibat

ALGS is a multisystemic disorder characterized by the buildup of bile in the liver causing damage. Symptoms include pruritus, jaundice, xanthomas, darkening of the urine, light-colored, loose, greasy, and odorous stools, hepatomegaly, and splenomegaly.

Odevixibat is an experimental treatment being developed by Albireo Pharma. It is a nonsystemic ileal bile acid transport inhibitor, which works by decreasing the reabsorption of bile acids from the small intestine to the liver. The treatment could reduce the symptoms of the disease and delay or prevent hepatic damage caused by toxic levels of bile acid accumulation.

The European Medicines Agency granted odevixibat Orphan Drug Designation for the treatment of ALGS, biliary atresia, and primary biliary cholangitis. 

Reference

Long-term safety and efficacy of odevixibat in patients with Alagille syndrome (ASSERT-EXT). US National Library of Medicine. Updated March 10, 2022. Accessed March 23, 2022. 

Albireo Pharma ASSERT-EXT biliary atresia Bylvay Gastroenterologists Hepatologists Nephrologists odevixibat Physicians primary biliary cholangitis Researchers