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Can a daily pill help kids with rare liver disease avoid transplant?

NCT ID NCT06850038

Disease control Sponsor: Ipsen Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 01, 2026 · Updated 5 times

Summary

This study follows 30 people with Alagille syndrome who are taking odevixibat (Bylvay) in their daily lives. The goal is to see if the drug helps them avoid serious surgeries like liver transplant or bile duct repair over the long term. Researchers will also track weight, height, and safety. Because it's an observational registry, it reflects real-world use but cannot prove the drug causes the outcomes.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
odevixibat (Bylvay)
What this could lead to
If this works, it could show that odevixibat helps children with Alagille syndrome avoid or delay the need for liver transplant or bile duct surgery.
What could go wrong
This is a small, observational registry (30 people) with no placebo group, so results may not prove the drug works better than no treatment. It also only follows people already taking the drug, which can bias outcomes.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 30 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2025

Expected to finish

Nov 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patients diagnosed with ALGS who start treatment with odevixibat (Bylvay) will be enrolled into the registry. Patients who started odevixibat treatment before the implementation of the registry study may also be enrolled.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Diagnosed with ALGS. * On (or starting) active odevixibat treatment. * Signed informed consent and assent, as appropriate. Consent/assent from the participant or legal representative should be obtained, as appropriate, before any study data collection is conducted. Participants who turn 18 years of age (or legal age per country) while participating in the study will be required to provide consent for themselves. Exclusion Criteria: * Currently participating in a clinical trial with odevixibat. * Currently participating in any interventional clinical trial for ALGS. * Have any contraindication to odevixibat as per the locally approved label. * Had liver transplant before enrolment

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Conditions

The condition(s) this trial relates to.

Alagille syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Alagille syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    10 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Children's Hospital Los Angeles

    RECRUITING

    Los Angeles, California, 90027, United States

  • Children's Medical Center Of Dallas

    RECRUITING

    Dallas, Texas, 75246, United States

  • Columbia University Medical Center - Morgan Stanley Children's Hospital of NewYork - Presbyterian (MS-CHONY)

    RECRUITING

    New York, New York, 10032, United States

  • Le Bonheur Children's Hospital

    RECRUITING

    Memphis, Tennessee, 38105, United States

  • NYU Langone - NYU Grossman School of Medicine

    RECRUITING

    New York, New York, 10016, United States

  • Primary Children's Hospital (PCH) - Intermountain Healthcare/University of Utah

    NOT_YET_RECRUITING

    Salt Lake City, Utah, 84113, United States

  • Proactive El Paso,LLC

    RECRUITING

    El Paso, Texas, 79902, United States

  • SSM Health Cardinal Glennon Children's Hospital

    RECRUITING

    St Louis, Missouri, 63104, United States

  • The Children's Mercy Hospital

    RECRUITING

    Kansas City, Missouri, 64108, United States

  • UCSF Benioff Children's Hospital

    RECRUITING

    San Francisco, California, 94158, United States

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