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)

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.

Study Overview

• Status: Recruiting
• Conditions: Alagille Syndrome

• Study Type: Observational
• Enrollment (Estimated): 30

Contacts and Locations

• Study Contact: Name: Ipsen Recruitment Enquiries; Phone Number: See email; Email: clinical.trials@ipsen.com

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90027
      • Recruiting
      • Children's Hospital Los Angeles
    • San Francisco, California, United States, 94158
      • Recruiting
      • UCSF Benioff Children's Hospital
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Recruiting
      • The Children's Mercy Hospital
    • St Louis, Missouri, United States, 63104
      • Not yet recruiting
      • SSM Health Cardinal Glennon Children's Hospital
  • New York
    • New York, New York, United States, 10016
      • Recruiting
      • NYU Langone - NYU Grossman School of Medicine
    • New York, New York, United States, 10032
      • Recruiting
      • Columbia University Medical Center - Morgan Stanley Children's Hospital of NewYork - Presbyterian (MS-CHONY)
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • Recruiting
      • Le Bonheur Children's Hospital
  • Texas
    • Dallas, Texas, United States, 75246
      • Recruiting
      • Children's Medical Center of Dallas
    • El Paso, Texas, United States, 79902
      • Recruiting
      • Proactive El Paso,LLC
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • Not yet recruiting
      • Primary Children's Hospital (PCH) - Intermountain Healthcare/University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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.

Description

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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation.		From first dose to end of study (approximately 5 years data collection)
Surgical biliary diversion-free survival	Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.	From first dose to end of study (approximately 5 years data collection)
Liver transplant-free survival	Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.	From first dose to end of study (approximately 5 years data collection)
Overall survival	Defined as time from the start of odevixibat treatment to death.	From first dose to end of study (approximately 5 years data collection)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline in Weight	This endpoint will measure the change in participants' weight from baseline, reported as standard-deviation scores (z-scores) to account for age and sex-specific growth variations.	From first dose to end of study (approximately 5 years data collection)
Change from Baseline in Height	This endpoint will measure the change in participants' height from baseline, reported as standard-deviation scores (z-scores) to account for age and sex-specific growth variations.	From first dose to end of study (approximately 5 years data collection)
Percentage of participants with Adverse Events (AEs) associated with fat-soluble vitamin (FSV) deficiencies and their possible sequelae.	An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.	From signing of the Informed Consent Form (ICF) to 180 days after the last dose intake of odevixibat
Percentage of participants with suspected hepatotoxic Adverse events (AEs) requiring interruption of odevixibat	An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.	From signing of the ICF to 180 days after the last dose intake of odevixibat
Percentage of participants with bleeding AEs	An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.	From signing of the ICF to 180 days after the last dose intake of odevixibat
Percentage of participants with AEs	An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.	From signing of the ICF to 180 days after the last dose intake of odevixibat

Collaborators and Investigators

• Sponsor: Ipsen
• Investigators: Study Director: Ipsen Medical, Director, Ipsen

Study record dates

Study Major Dates

• Study Start (Actual): April 22, 2025
• Primary Completion (Estimated): November 1, 2029
• Study Completion (Estimated): November 1, 2029

Study Registration Dates

• First Submitted: February 13, 2025
• First Submitted That Met QC Criteria: February 26, 2025
• First Posted (Actual): February 27, 2025

Study Record Updates

• Last Update Posted (Actual): May 28, 2026
• Last Update Submitted That Met QC Criteria: May 27, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Genetic Diseases, Inborn; Digestive System Diseases; Biliary Tract Diseases; Liver Diseases; Congenital Abnormalities; Cardiovascular Abnormalities; Heart Defects, Congenital; Abnormalities, Multiple; Bile Duct Diseases; Cholestasis, Intrahepatic; Cholestasis; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Alagille Syndrome
• Other Study ID Numbers: CLIN-60240-033

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.

Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.

• IPD Sharing Time Frame: Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
• IPD Sharing Access Criteria: Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No