Alagille SyndromeNCT06850038
A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
An observational study of Alagille Syndrome, run by Ipsen at 10 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Alagille Syndrome
- How many people
- 30 participants
- Where
- 10 sitesUnited States
- Study dates
- 22 Apr 2025to 30 Nov 2030
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
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.
- Had liver transplant before enrolment
Trial locations and contacts
10 sites in 1 country. Contact the study team or a site near you to ask about taking part.
10 sites in 1 country
United States10 sites, 10 recruiting
- Recruiting
Children's Medical Center Of Dallas
Dallas, Texas, 75246
- Recruiting
Proactive El Paso,LLC
El Paso, Texas, 79902
- Recruiting
The Children's Mercy Hospital
Kansas City, Missouri, 64108
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
- Recruiting
Le Bonheur Children's Hospital
Memphis, Tennessee, 38105
- Recruiting
NYU Langone - NYU Grossman School of Medicine
New York, New York, 10016
- Recruiting
Columbia University Medical Center - Morgan Stanley Children's Hospital of NewYork - Presbyterian (MS-CHONY)
New York, New York, 10032
- Not yet recruiting
Primary Children's Hospital (PCH) - Intermountain Healthcare/University of Utah
Salt Lake City, Utah, 84113
- Recruiting
UCSF Benioff Children's Hospital
San Francisco, California, 94158
- Recruiting
SSM Health Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104
About this study
Official title: 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.
- 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 with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From first dose to end of study (approximately 5 years data collection)
Secondary outcomes (6)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From signing of the ICF to 180 days after the last dose intake of odevixibat
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2025
- Study start22 Apr 2025
- Primary completion30 Nov 2030 (estimated)
- Study completion30 Nov 2030 (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
- NCT06850038
- Sponsor's protocol ID
- CLIN-60240-033
- Status last verified
- Oct 2026
- First submitted
- 13 Feb 2025
- 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.