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Efficacy and Safety Evaluation of Odevixibat in Pediatric Biliary Atresia Post-Kasai Hepatoportoenterostomy: A Double-Blind, Randomized, Placebo-Controlled Trial

Fast replyInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Biliary Atresia, a condition affecting the liver and bile ducts in children. The study will evaluate the effectiveness and safety of a medication called Odevixibat (also known by its code name A4250). This medication is taken in the form of a capsule and is designed to help improve liver function in children who have undergone a surgical procedure called Kasai Hepatoportoenterostomy, which is performed to treat Biliary Atresia.

The purpose of the study is to compare the effects of Odevixibat with a placebo in children with Biliary Atresia. Participants will receive either Odevixibat or a placebo once daily. The study will last for up to 104 weeks, during which time the health of the participants will be closely monitored. The main focus will be on the time it takes for participants to require a liver transplant or experience other serious health issues related to their liver condition.

Throughout the study, various health parameters will be assessed, including liver function tests and overall health status. The study aims to provide valuable information on whether Odevixibat can help improve the outcomes for children with Biliary Atresia who have had the Kasai procedure. Participants will be regularly checked for any side effects or changes in their health to ensure their safety during the trial.

The research process

The trial runs in 8 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization

    After joining the study, the patient is assigned by a computer system to receive either odevixibat or a matching placebo. the assignment is double‑blind, meaning neither the patient nor the study staff knows which treatment is given.

  2. Step 2

    Baseline assessment

    A series of evaluations is performed before the first dose. these include a physical examination, collection of medical history, blood tests for total bilirubin, serum bile acids, vitamins and lipids, and an abdominal ultrasound. the purpose is to document the patient’s condition at the start of the trial.

  3. Step 3

    Start of medication

    The patient begins taking one oral capsule each day. the dose is calculated as 120 µg per kilogram of body weight. the capsule is taken with food and the schedule is maintained for up to 104 weeks.

  4. Step 4

    Daily dosing

    The patient continues the once‑daily capsule throughout the treatment period. consistency is important; missed doses should be avoided.

  5. Step 5

    Scheduled clinic visits

    Clinic visits are planned at week 13, week 26, week 52, and week 104. at each visit the patient undergoes blood tests to measure total bilirubin and serum bile acid levels, a physical examination, and an abdominal ultrasound. these assessments monitor the effect of the medication and the safety of the patient.

    The visits also provide an opportunity to discuss any side effects or concerns with the study physician.

  6. Step 6

    Safety monitoring

    Throughout the trial the patient is asked to report any new symptoms or adverse events. the study team records these events and may perform additional laboratory tests or imaging if needed.

  7. Step 7

    Possible early termination

    If the patient experiences a liver transplant or death, the treatment period ends for that individual and the event is recorded as the primary outcome of the study.

  8. Step 8

    Final assessment

    At week 104 the patient completes the last set of evaluations, which include the same laboratory tests and imaging performed at earlier visits. the results determine whether the patient remained alive without a liver transplant during the 104‑week period.

Who can join the trial?

3 criteria

  • A boy or girl who has been told by a doctor that they have biliary atresia (a rare liver condition that blocks the flow of bile).
  • The child was 90 days old or younger when they had the Kasai hepatoportoenterostomy (HPE) surgery, which is an operation to help the liver drain bile.
  • The child can begin taking the study medicine no later than three weeks after the Kasai HPE surgery.

Who cannot join the trial?

9 criteria

  • Having fluid buildup in the belly that cannot be controlled, called intractable ascites.
  • Having had surgery that removed a part of the small intestine called the ileum, known as ileal resection surgery.
  • Having a liver blood test called ALT that is 10 times higher than the normal upper limit, which suggests serious liver injury.
  • Needing nutrition delivered through a vein (total parenteral nutrition) or being unable to swallow the study medicine, at the time of joining the trial.
  • Having a sudden infection of the bile ducts, called acute ascending cholangitis, unless it has completely cleared.
  • Having a birth‑related condition that forms cysts in the bile duct, known as choledochal cystic disease.
  • Having a blood clotting test result (INR) higher than 1.6, which means blood clots slowly; this can sometimes be lowered with a vitamin called Vitamin K.
  • Having any other health problems—such as birth defects, major heart surgery, serious liver, bile‑duct, or digestive‑tract disease—that the doctor thinks could make the study unsafe or affect the results.
  • Weighing less than 3.5 kilograms (about 7.7 pounds) at the time of joining the trial.
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Investigated drugs

A4250 is an oral capsule that contains the medicine odevixibat. It is being studied as a treatment for children who have biliary atresia, a liver condition, after they have had a Kasai hepatoportoenterostomy surgery. In the trial, participants take the capsule once each day for up to two years. Researchers are looking at how well the medicine helps the children’s own liver keep working (native liver survival) and whether it is safe to use over a long period. The drug is classified as an orphan drug, meaning it is intended for a rare disease.

What is already known about the treatment

Odevixibat - Odevixibat is taken as a small capsule that is swallowed once a day, with the dose calculated by the child’s weight (about 120 µg per kilogram). It is an orphan‑drug that has been approved in several countries for rare liver disorders and is being studied for biliary atresia, a serious newborn liver disease. The medicine works by blocking a protein in the intestine that normally re‑absorbs bile acids, so fewer bile acids return to the liver, which helps reduce liver damage. It belongs to the class of ileal bile‑acid transporter inhibitors, a type of oral therapy for cholestatic conditions.

Investigated diseases

Biliary Atresia - Biliary Atresia is a birth‑related disorder in which the tubes that carry bile from the liver to the intestine are missing or become blocked. Because bile cannot flow out, it builds up in the liver, causing yellowing of the skin and eyes, and swelling of the abdomen. The condition usually appears within the first weeks of life and worsens as the trapped bile damages liver tissue. Over months, the liver may become scarred and less able to function properly. This gradual loss of liver function can continue throughout childhood.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2024-512086-14-00Protocol codeA4250-011Estimated enrolment243 patientsSponsorIpsen Pharma

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

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