Menu
Recruiting NCT07585097

A Study to Observe the Long-term Safety of Odevixibat 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: from 6 months · 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
France, Italy
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 Non-Interventional Study Evaluating the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS)

Overview

This study will collect information from patients with ALGS who are using odevixibat in their daily lives. Odevixibat is a medication that helps patients with ALGS, a rare disease that affects the liver and causes itching. The main aim of this study is to observe the long-term, everyday safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.

Primary outcome measures

  • Percentage of participants experiencing adverse events (AEs) [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Percentage of participants experiencing serious adverse events (SAEs) [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
Secondary outcome measures (12)
  • Percentage of participants with severe diarrhoea events [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Percentage of participants with bloody diarrhoea events [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Percentage of participants experiencing diarrhoea events with concurrent dehydration [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Percentage of participants experiencing diarrhoea events treated with oral or intravenous rehydration [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Change from baseline in fat-soluble vitamin (FSV) levels [Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)]
  • Percentage of participants with fat-soluble vitamin deficiency [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Percentage of participants with clinical manifestations of fat-soluble vitamin deficiency [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Percentage of participants with suspected hepatotoxicity requiring interruption of odevixibat treatment [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Percentage of participants with clinical manifestations related to hepatotoxicity [Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)]
  • Change from baseline in alanine aminotransferase (ALT) [Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)]
  • Change from baseline in aspartate aminotransferase (AST) [Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)]
  • Change from baseline in gamma-glutamyl transferase (GGT) [Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)]

Eligibility criteria

Inclusion criteria

  • Diagnosed with ALGS.
  • On (or starting) active odevixibat treatment.
  • Aged 6 months or older at the time of consent.

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.

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

France · 8 centers
  • Hospices Civils de Lyon - Hopital Femme Mere Enfant — Bron
  • CHU Dijon Bourgogne — Dijon
  • APHP - Hopital Bicetre - Paris Sud — Le Kremlin-Bicêtre
  • APHM - CHU Timone — Marseille
  • CHU de Montpellier - Hopital Saint Eloi — Montpellier
  • APHP - Hopital Necker-Enfants Malades — Paris
  • CHU de Toulouse - Hopital Paule de Viguier — Toulouse
  • APHP - Centre Hepato-Biliaire Hospital Paul Brousse — Villejuif
Italy · 6 centers
  • Azienda Ospedaliero Universitaria Meyer — Florence
  • Azienda Ospedaliera Di Rilievo Nazionale Santobono Pausilipon — Naples
  • University Hospital Of Padova — Padova
  • IRCCS Ospedale Pediatrico Bambino Gesu — Roma
  • A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita — Torino
  • Ospedale Universitario Di Udine — Udine

Identifiers

NCT: NCT07585097 · CLIN-60240-034

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗