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)
1 other identifier
observational
30
1 country
9
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Apr 2025
Longer than P75 for all trials
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 13, 2025
CompletedFirst Posted
Study publicly available on registry
February 27, 2025
CompletedStudy Start
First participant enrolled
April 22, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
November 1, 2029
April 30, 2026
April 1, 2026
4.5 years
February 13, 2025
April 29, 2026
Conditions
Outcome Measures
Primary Outcomes (4)
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 Outcomes (6)
Change from Baseline in Weight
From first dose to end of study (approximately 5 years data collection)
Change from Baseline in Height
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.
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
From signing of the ICF to 180 days after the last dose intake of odevixibat
Percentage of participants with bleeding AEs
From signing of the ICF to 180 days after the last dose intake of odevixibat
- +1 more secondary outcomes
Eligibility Criteria
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.
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Ipsenlead
Study Sites (9)
Childrens Hospital Los Angeles
Los Angeles, California, 90027, United States
UCSF Pediatric Gastroenterology
San Francisco, California, 94158, United States
The Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
St Louis University. SSM Cardinal Glennon Childrens Medical Center
St Louis, Missouri, 63104, United States
Columbia University
New York, New York, 10016, United States
NYU Langone - NYU Grossman School of Medicine
New York, New York, 10016, United States
Le Bonheur Children's Hospital
Memphis, Tennessee, 38105, United States
UT Southwestern Childrens Medical Center
Dallas, Texas, 75235, United States
Proactive El Paso,LLC
El Paso, Texas, 79902, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Ipsen Medical, Director
Ipsen
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- OTHER
- Time Perspective
- PROSPECTIVE
- Target Duration
- 2 Years
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 13, 2025
First Posted
February 27, 2025
Study Start
April 22, 2025
Primary Completion (Estimated)
November 1, 2029
Study Completion (Estimated)
November 1, 2029
Last Updated
April 30, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will share
- 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.
- 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/).
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.