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A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment

28 août 2026 mis à jour par: Ipsen

Prospective Non-Interventional Study Evaluating the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS)

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.

Aperçu de l'étude

Statut

Recrutement

Les conditions

Type d'étude

Observationnel

Inscription (Estimé)

30

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Coordonnées de l'étude

Lieux d'étude

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

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

  • Enfant
  • Adulte
  • Adulte plus âgé

Accepte les volontaires sains

Non

Méthode d'échantillonnage

Échantillon non probabiliste

Population étudiée

Participants with Alagille syndrome (ALGS) who have been prescribed odevixibat by their treating physician will be enrolled into the study. Participants who started odevixibat treatment before study implementation may also be enrolled.

La description

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.

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Percentage of participants experiencing adverse events (AEs)
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment.
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)
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment.
From first ICF signature and up to end of data collection (approximately 5 years of data collection)

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Percentage of participants with severe diarrhoea events
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with bloody diarrhoea events
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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
Délai: From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with fat-soluble vitamin deficiency
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
For example, bleeding, rickets, or osteopenia.
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
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in alanine aminotransferase (ALT)
Délai: From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in aspartate aminotransferase (AST)
Délai: From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in gamma-glutamyl transferase (GGT)
Délai: From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in blood bilirubin
Délai: From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in international normalized ratio (INR)
Délai: From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to diarrhoea
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to hepatotoxicity
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to fat-soluble vitamin deficiency
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with treatment discontinuations due to diarrhoea
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection).
From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with treatment discontinuations due to hepatotoxicity
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection).
From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with treatment discontinuations due to fat-soluble vitamin deficiency
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection).
From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with pregnancy and maternal complications
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of foetuses, neonates, or infants with adverse effects following exposure to odevixibat during pregnancy and/or lactation
Délai: From first documented exposure during pregnancy or lactation and up to end of data collection (approximately 5 years of data collection)
From first documented exposure during pregnancy or lactation and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with biliary diversion surgery
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with liver transplantation
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants who die from any cause
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants switching from odevixibat to maralixibat
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Ipsen

Les enquêteurs

  • Directeur d'études: Ipsen Medical Director, Ipsen

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

10 juillet 2026

Achèvement primaire (Estimé)

30 septembre 2031

Achèvement de l'étude (Estimé)

30 septembre 2031

Dates d'inscription aux études

Première soumission

6 mai 2026

Première soumission répondant aux critères de contrôle qualité

6 mai 2026

Première publication (Réel)

13 mai 2026

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

31 août 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

28 août 2026

Dernière vérification

1 août 2026

Plus d'information

Termes liés à cette étude

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • CLIN-60240-034

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

OUI

Description du régime IPD

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.

Délai de partage IPD

Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.

Critères d'accès au partage IPD

Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Non

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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