RecruitingNCT06850038

A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment

Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)


Sponsor

Ipsen

Enrollment

30 participants

Start Date

Apr 22, 2025

Study Type

OBSERVATIONAL

Conditions

Summary

This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives. Odevixibat is a medicine that helps patients with ALGS, a rare disease that harms their liver and causes itching. The main aim of this study is to observe the long-term, everyday effectiveness and safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.


Eligibility

Inclusion Criteria3

  • 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 Criteria4

  • 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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Locations(9)

Childrens Hospital Los Angeles

Los Angeles, California, United States

UCSF Pediatric Gastroenterology

San Francisco, California, United States

The Children's Mercy Hospital

Kansas City, Missouri, United States

St Louis University. SSM Cardinal Glennon Childrens Medical Center

St Louis, Missouri, United States

Columbia University

New York, New York, United States

NYU Langone - NYU Grossman School of Medicine

New York, New York, United States

Le Bonheur Children's Hospital

Memphis, Tennessee, United States

UT Southwestern Childrens Medical Center

Dallas, Texas, United States

Proactive El Paso,LLC

El Paso, Texas, United States

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NCT06850038


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