Clinical trials are an important tool in medical research to understand more about the safety, efficacy, and tolerability of therapeutics – and how these may influence patient outcomes. Recently, biopharmaceutical company CANbridge Pharmaceuticals, Inc. (“CANbridge”) launched the Phase 2 EMBARK trial in China. This trial is designed to evaluate CAN108 (maralixibat) for those with biliary atresia who have undergone the Kasai procedure. According to a news release from July 14, the first trial participant was recently dosed.
Altogether, this trial aims to enroll around 92 patients with biliary atresia. Over a 26-week period, and 78-week extension period, researchers will evaluate the safety, efficacy, and tolerability of CAN108, which CANbridge describes as:
an orally administered small molecule ASBT inhibitor.
Outside of biliary atresia, CANbridge is also exploring CAN108 as a potential therapy for patients with progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome.
What is Biliary Atresia?
Biliary atresia is a rare condition of the liver and bile ducts in infants. Normally, the bile ducts carry bile from the liver to the gallbladder. However, biliary atresia is characterized by the scarring, destruction, and blocking of bile ducts. This causes bile to accumulate in the liver, causing scarring and other health issues. Doctors are not entirely sure what causes this condition. Some researchers hypothesize that something happens which causes the bile ducts to be damaged either in the womb or shortly following birth. This condition is more common in females than males.
Typically, symptoms appear within the first 2-8 weeks following birth. These symptoms can include:
- Jaundice (yellowing of the skin, eyes, and mucuous membranes)
- Dark urine and pale (yellow/white/gray) stool
- Irritability
- Poor weight gain or unintended weight loss
- Heightened blood pressure
- Enlarged liver and/or spleen
- Note: In regards to the spleen, those with this condition may also not have a spleen or have multiple spleens.
- Abdominal distention
- Heart and kidney abnormalities
- Itchiness
- Liver scarring
- Loss of liver function / liver failure
