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Recruiting NCT06850038

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

— Observational Alagille Syndrome

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Alagille Syndrome. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)

Overview

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.

Primary outcome measures

  • 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)]
  • Liver transplant-free survival [Time frame: From first dose to end of study (approximately 5 years data collection)]
  • Overall survival [Time frame: From first dose to end of study (approximately 5 years data collection)]
Secondary outcome measures (6)
  • 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]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Other

Study locations

United States · 10 centers
  • Children's Hospital Los Angeles — Los Angeles
  • UCSF Benioff Children's Hospital — San Francisco
  • The Children's Mercy Hospital — Kansas City
  • SSM Health Cardinal Glennon Children's Hospital — St Louis
  • NYU Langone - NYU Grossman School of Medicine — New York
  • Columbia University Medical Center - Morgan Stanley Children's Hospital of NewYork - Presb — New York
  • Le Bonheur Children's Hospital — Memphis
  • Children's Medical Center Of Dallas — Dallas
  • … and 2 more centers

Identifiers

NCT: NCT06850038 · CLIN-60240-033

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗