According to a recent press release from Albireo Pharma, the first participant has enrolled in the BOLD trial. The BOLD trial, which stands for Biliary atresia and the use of Odevixibat in treating Liver Disease, is a phase 3 trial designed to test the safety, tolerability, and efficacy of odevixibat.
Phase 3 BOLD Trial
The phase 3 BOLD trial will examine odevixibat as a potential therapeutic for patients with biliary atresia. Currently, there are no treatments for the condition. Thus, odevixibat could meet an unmet need within this patient population.
Within the study, 200 patients will either receive odevixibat at 120 μg/kg per day or a placebo. The entire study will last for approximately 2 years. A Kasai procedure is a necessary requirement for enrollment. Ultimately, the study seeks to understand whether odevixibat can prevent patients from requiring a liver transplant, as well as to test mortality rates, quality of life, and bilirubin and serum bile acid levels.
Odevixibat, which received Orphan Drug and Rare Pediatric Disease Designations, is a bile acid transporter inhibitor. It is designed to treat pediatric patients with liver disease, such as Alagille syndrome or biliary atresia.
Biliary Atresia
Biliary atresia is a rare liver condition that occurs in infants. Normally, bile ducts carry bile to the gallbladder to help with digestion. However, patients with biliary atresia experience bile duct scarring, damage, or blockages. As a result, bile accumulates, causing liver problems such as cirrhosis. While infants born with this condition may appear healthy at birth, symptoms appear within 2-3 weeks. Generally, the condition affects females more than males. Around 70% of all infant patients require a liver transplant.
Symptoms include:
- Swollen stomach
- Dark urine
- White, gray, or pale yellow stool
- Jaundice
- Liver enlargement and scarring
- Irritability
- Liver failure
- Intense itching
- Heart and kidney malformations
- Poor weight gain
Learn more about biliary atresia here.
