RecruitingNot Applicable

A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment

United States30 participantsStarted 2025-04-22

Plain-language 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.

Who can participate

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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. Exclusion Criteria: * 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

What they're measuring

Percentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation.

Timeframe: From first dose to end of study (approximately 5 years data collection)

Surgical biliary diversion-free survival

Timeframe: From first dose to end of study (approximately 5 years data collection)

Liver transplant-free survival

Timeframe: From first dose to end of study (approximately 5 years data collection)

Overall survival

Timeframe: From first dose to end of study (approximately 5 years data collection)

Trial details

NCT IDNCT06850038

SponsorIpsen

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2029-11

Contact for this trial

Ipsen Recruitment Enquiries

See email clinical.trials@ipsen.com

View on ClinicalTrials.gov More Alagille Syndrome trials

Plain-language summary

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Percentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation.

Timeframe: From first dose to end of study (approximately 5 years data collection)

Surgical biliary diversion-free survival

Timeframe: From first dose to end of study (approximately 5 years data collection)

Liver transplant-free survival

Timeframe: From first dose to end of study (approximately 5 years data collection)

Overall survival

Timeframe: From first dose to end of study (approximately 5 years data collection)