A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
Prospective Non-Interventional Study Evaluating the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS)
1 other identifier
observational
30
2 countries
14
Brief Summary
This study will collect information from patients with ALGS who are using odevixibat in their daily lives. Odevixibat is a medication that helps patients with ALGS, a rare disease that affects the liver and causes itching. The main aim of this study is to observe the long-term, everyday 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 Jul 2026
Longer than P75 for all trials
14 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
May 6, 2026
CompletedFirst Posted
Study publicly available on registry
May 13, 2026
CompletedStudy Start
First participant enrolled
July 10, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 30, 2031
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 30, 2031
October 1, 2026
September 1, 2026
5.2 years
May 6, 2026
September 29, 2026
Conditions
Outcome Measures
Primary Outcomes (2)
Percentage of participants experiencing adverse events (AEs)
An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment.
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants experiencing serious adverse events (SAEs)
An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment.
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Secondary Outcomes (26)
Percentage of participants with severe diarrhoea events
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with bloody diarrhoea events
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants experiencing diarrhoea events with concurrent dehydration
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants experiencing diarrhoea events treated with oral or intravenous rehydration
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in fat-soluble vitamin (FSV) levels
From baseline and up to end of data collection (approximately 5 years of data collection)
- +21 more secondary outcomes
Eligibility Criteria
Participants with Alagille syndrome (ALGS) who have been prescribed odevixibat by their treating physician will be enrolled into the study. Participants who started odevixibat treatment before study implementation may also be enrolled.
You may qualify if:
- Diagnosed with ALGS.
- On (or starting) active odevixibat treatment.
- Aged 6 months or older at the time of consent.
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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Ipsenlead
Study Sites (14)
Hospices Civils de Lyon - Hopital Femme Mere Enfant
Bron, France
CHU Dijon Bourgogne
Dijon, France
APHP - Hopital Bicetre - Paris Sud
Le Kremlin-Bicêtre, France
APHM - CHU Timone
Marseille, France
CHU de Montpellier - Hopital Saint Eloi
Montpellier, France
APHP - Hopital Necker-Enfants Malades
Paris, France
CHU de Toulouse - Hopital Paule de Viguier
Toulouse, France
APHP - Centre Hepato-Biliaire Hospital Paul Brousse
Villejuif, France
Azienda Ospedaliero Universitaria Meyer
Florence, Italy
Azienda Ospedaliera Di Rilievo Nazionale Santobono Pausilipon
Naples, Italy
University Hospital Of Padova
Padova, Italy
IRCCS Ospedale Pediatrico Bambino Gesu
Roma, Italy
A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita
Torino, Italy
Ospedale Universitario Di Udine
Udine, Italy
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
May 6, 2026
First Posted
May 13, 2026
Study Start
July 10, 2026
Primary Completion (Estimated)
September 30, 2031
Study Completion (Estimated)
September 30, 2031
Last Updated
October 1, 2026
Record last verified: 2026-09
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.