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
- Are there results?
- Not yet: the study is under way
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.
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)
- ClinicalTrials.gov ID
- NCT06850038
- Acronym / sponsor ID
- CLIN-60240-033
- Conditions
- Alagille Syndrome
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- 22 Apr 2025 (actual)
- Primary completion
- 30 Nov 2030 (estimated)
- Study completion
- 30 Nov 2030 (estimated)
- First posted
- 27 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Other
- Time perspective
- Prospective
- Target follow-up
- 2 Years
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- 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.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Percentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation.
Time frame: From first dose to end of study (approximately 5 years data collection)
Surgical biliary diversion-free survival
Time frame: From first dose to end of study (approximately 5 years data collection)
Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
Liver transplant-free survival
Time frame: From first dose to end of study (approximately 5 years data collection)
Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
Overall survival
Time frame: From first dose to end of study (approximately 5 years data collection)
Defined as time from the start of odevixibat treatment to death.
6 secondary outcomes
Change from Baseline in Weight
Time frame: From first dose to end of study (approximately 5 years data collection)
Change from Baseline in Height
Time frame: 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.
Time frame: 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
Time frame: From signing of the ICF to 180 days after the last dose intake of odevixibat
Percentage of participants with bleeding AEs
Time frame: From signing of the ICF to 180 days after the last dose intake of odevixibat
Percentage of participants with AEs
Time frame: From signing of the ICF to 180 days after the last dose intake of odevixibat
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2026.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ipsen Medical, Director study director
Ipsen
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alagille Syndrome, run by Ipsen at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ipsen Recruitment Enquiries (See email, clinical.trials@ipsen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation. (time frame: From first dose to end of study (approximately 5 years data collection)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Oct 2026.