Alagille SyndromeNCT07585097
A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
An observational study of Alagille Syndrome, run by Ipsen at 14 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Alagille Syndrome
- How many people
- 30 participants
- Where
- 14 sitesin 2 countries
- Study dates
- 10 Jul 2026to 30 Sep 2031
- How long will I take part?
- 2 Years
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 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.
Who may be able to join
- Diagnosed with ALGS.
- On (or starting) active odevixibat treatment.
- Aged 6 months or older at the time of consent.
Who cannot join
- 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.
Trial locations and contacts
14 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
14 sites in 2 countries
France8 sites, 8 recruiting
- Recruiting
Hospices Civils de Lyon - Hopital Femme Mere Enfant
Bron
- Not yet recruiting
CHU Dijon Bourgogne
Dijon
- Not yet recruiting
APHP - Hopital Bicetre - Paris Sud
Le Kremlin-Bicêtre
- Not yet recruiting
APHM - CHU Timone
Marseille
- Not yet recruiting
CHU de Montpellier - Hopital Saint Eloi
Montpellier
- Not yet recruiting
APHP - Hopital Necker-Enfants Malades
Paris
- Not yet recruiting
CHU de Toulouse - Hopital Paule de Viguier
Toulouse
- Not yet recruiting
APHP - Centre Hepato-Biliaire Hospital Paul Brousse
Villejuif
Italy6 sites, 6 recruiting
- Recruiting
Azienda Ospedaliero Universitaria Meyer
Florence
- Recruiting
Azienda Ospedaliera Di Rilievo Nazionale Santobono Pausilipon
Naples
- Not yet recruiting
University Hospital Of Padova
Padova
- Not yet recruiting
IRCCS Ospedale Pediatrico Bambino Gesu
Roma
- Not yet recruiting
A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita
Torino
- Not yet recruiting
Ospedale Universitario Di Udine
Udine
About this study
Official title: Prospective Non-Interventional Study Evaluating the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS)
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.
- Conditions
- Alagille Syndrome
- Medical subject headings
- Alagille Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with bloody diarrhoea events
Measured: 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
Measured: 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
Measured: 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
Measured: From baseline and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with fat-soluble vitamin deficiency
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with clinical manifestations of fat-soluble vitamin deficiency
For example, bleeding, rickets, or osteopenia.
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with suspected hepatotoxicity requiring interruption of odevixibat treatment
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with clinical manifestations related to hepatotoxicity
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in alanine aminotransferase (ALT)
Measured: From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in aspartate aminotransferase (AST)
Measured: From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in gamma-glutamyl transferase (GGT)
Measured: From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in blood bilirubin
Measured: From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in international normalized ratio (INR)
Measured: From baseline and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to diarrhoea
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to hepatotoxicity
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to fat-soluble vitamin deficiency
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with treatment discontinuations due to diarrhoea
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with treatment discontinuations due to hepatotoxicity
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with treatment discontinuations due to fat-soluble vitamin deficiency
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with pregnancy and maternal complications
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of foetuses, neonates, or infants with adverse effects following exposure to odevixibat during pregnancy and/or lactation
Measured: From first documented exposure during pregnancy or lactation and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with biliary diversion surgery
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with liver transplantation
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants who die from any cause
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants switching from odevixibat to maralixibat
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2026
- Study start10 Jul 2026
- Primary completion30 Sep 2031 (estimated)
- Study completion30 Sep 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alagille SyndromeStudies of the same condition, open and under way.
- More trials run by IpsenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 30 participants (estimated)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07585097
- Sponsor's protocol ID
- CLIN-60240-034
- Status last verified
- Oct 2026
- First submitted
- 6 May 2026
- Last updated
- 23 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.