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Long-term Study on the Safety and Effectiveness of Odevixibat for Patients with Alagille Syndrome

Verified siteRegistered drugNo 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 the long-term safety and effectiveness of a medication called odevixibat (also known by its code name A4250) in patients with Alagille syndrome. Alagille syndrome is a genetic disorder that can affect the liver, heart, and other parts of the body, often leading to symptoms like severe itching. The purpose of this study is to see how well odevixibat works over an extended period and to ensure it is safe for patients to use.

The study involves two groups of patients. The first group includes patients who have already completed a previous study with odevixibat and will continue to take the medication to see if it helps with itching. The second group consists of younger patients, under 12 months old, who will be monitored to evaluate the safety and how well they tolerate the medication. Participants will take odevixibat in capsule form by mouth, and the study will last up to 72 weeks.

Throughout the study, researchers will observe changes in symptoms such as itching and scratching, as well as other factors like sleep quality and overall quality of life. The study aims to provide valuable information on how odevixibat can help manage symptoms of Alagille syndrome over a long period, contributing to better treatment options for those affected by this condition.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after completing the 24-week treatment period of a previous study, A4250-012, for those in Cohort 1. Infants in Cohort 2 must have a confirmed diagnosis of Alagille syndrome and meet specific age and weight criteria.

  2. Step 2

    Medication administration

    The medication used in this study is odevixibat, also known as A4250. It is administered in the form of a capsule for oral use. The dosage and frequency are determined by the study protocol and are specific to each participant's needs.

  3. Step 3

    Monitoring and assessments

    Participants are monitored for changes in symptoms such as itching and scratching, using caregiver-reported instruments. These assessments occur regularly throughout the study, up to Week 72.

    Additional evaluations include changes in serum bile acid levels, sleep patterns, and quality of life scores. These are measured at various intervals, including Weeks 4, 12, 24, 48, and 72.

  4. Step 4

    Completion of the study

    The study is expected to conclude by April 16, 2027. Participants will have completed all required assessments and medication administration by this time.

Who can join the trial?

8 criteria

  • For Cohort 1: You must have completed the 24-week Treatment Period of Study A4250-012.
  • For Cohort 1: You need to sign an informed consent form. If you turn 18 years old during the study, you will need to sign again to continue participating.
  • For Cohort 1: You and your caregiver must be willing and able to use an electronic diary device as required by the study.
  • For Cohort 1: If you are sexually active, you must agree to use a reliable method of birth control, like an intra-uterine device or complete abstinence, from the time you sign the consent form until 90 days after your last dose of the study drug.
  • For Cohort 2: You must be an infant with clinically confirmed **Alagille Syndrome** and be 11 months old or younger at the start of the study.
  • For Cohort 2: You must weigh at least 2 kg at the start of the study.
  • For Cohort 2: You must have been born at a gestational age of at least 36 weeks. If you were born between 32 and 36 weeks, your postmenstrual age must be at least 36 weeks.
  • For Cohort 2: Your parent or legal guardian must sign an informed consent form for you to participate.

Who cannot join the trial?

6 criteria

  • Patients who have not completed the previous study called A4250-012 (ASSERT).
  • Patients who are not diagnosed with Alagille Syndrome, a genetic disorder that can affect the liver, heart, and other parts of the body.
  • Patients who are younger than 12 months old for certain parts of the study.
  • Patients who are not within the specified age range for the study.
  • Patients who are not able to follow the study procedures or take the study medication as required.
  • Patients who have any other medical condition that might interfere with the study or pose a risk to the patient.
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Investigated drugs

Odevixibat is a medication being studied for its long-term safety and effectiveness in treating patients with Alagille Syndrome. This condition can cause severe itching, and the study aims to see if odevixibat can help reduce this symptom over time. The trial also looks at how safe and tolerable the medication is for young patients under 12 months old.

What is already known about the treatment

Odevixibat – This medication is administered orally and is currently being studied for its long-term safety and effectiveness in treating Alagille Syndrome, a rare genetic disorder. It is in the clinical trial phase, with ongoing research to confirm its benefits and safety profile. Odevixibat is primarily indicated for reducing pruritus, or severe itching, in patients with Alagille Syndrome. At the molecular level, it works by inhibiting the ileal bile acid transporter, which reduces the reabsorption of bile acids and helps alleviate symptoms. It is classified as a bile acid transporter inhibitor.

Investigated diseases

Alagille Syndrome – Alagille Syndrome is a genetic disorder that primarily affects the liver, heart, and other parts of the body. It is characterized by a reduced number of bile ducts in the liver, leading to bile buildup and liver damage. This can cause symptoms such as jaundice, itching, and poor growth in children. The condition may also involve heart defects, distinctive facial features, and abnormalities in the eyes, spine, and kidneys. The severity and range of symptoms can vary widely among individuals. Over time, the liver and other affected organs may experience progressive damage.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-509028-17-00Protocol codeA4250-015Estimated enrolment60 patientsSponsorIpsen Pharma

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