Clinical trial NCT06850038: A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
Official title: Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Alagille Syndrome
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2025-02-27
Enrollment: 30
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: 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)
measure: Surgical biliary diversion-free survival
timeFrame: From first dose to end of study (approximately 5 years data collection)
measure: Liver transplant-free survival
timeFrame: From first dose to end of study (approximately 5 years data collection)
measure: Overall survival
timeFrame: From first dose to end of study (approximately 5 years data collection)
Secondary outcomes:
measure: Change from Baseline in Weight
timeFrame: From first dose to end of study (approximately 5 years data collection)
measure: Change from Baseline in Height
timeFrame: From first dose to end of study (approximately 5 years data collection)
measure: Percentage of participants with Adverse Events (AEs) associated with fat-soluble vitamin (FSV) deficiencies and their possible sequelae.
timeFrame: From signing of the Informed Consent Form (ICF) to 180 days after the last dose intake of odevixibat
measure: Percentage of participants with suspected hepatotoxic Adverse events (AEs) requiring interruption of odevixibat
timeFrame: From signing of the ICF to 180 days after the last dose intake of odevixibat
measure: Percentage of participants with bleeding AEs
timeFrame: From signing of the ICF to 180 days after the last dose intake of odevixibat
measure: Percentage of participants with AEs
timeFrame: From signing of the ICF to 180 days after the last dose intake of odevixibat
Other outcomes:
Locations:
city: New York
country: United States
facility: NYU Langone - NYU Grossman School of Medicine
state: New York
city: El Paso
country: United States
facility: Proactive El Paso,LLC
state: Texas
Study officials:
affiliation: Ipsen
name: Ipsen Medical, Director
role: STUDY_DIRECTOR
References: