A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China
28 août 2026 mis à jour par: Ipsen
Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay).
This registry-based study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) who take odevixibat (Bylvay) as part of routine clinical care in China.
PFIC is a rare genetic liver disease that affects bile secretion and can cause bile acids to build up in the liver, which may lead to symptoms such as severe itching (pruritus).
Odevixibat was first allowed to be used for PFIC in babies older than 6 months by the European Medicines Agency (EMA) on 16 July 2021 and by the United States Food and Drug Administration (FDA) on 20 July 2021 for itching in babies older than 3 months. Odevixibat is approved for the treatment of pruritus in PFIC and was approved in China on 01 December 2024 for patients 6 months of age and older with PFIC.
The main aim of this registry is to assess long-term real-world safety (based on adverse events) and to describe effectiveness outcomes.
Aperçu de l'étude
Statut
Recrutement
Les conditions
Type d'étude
Observationnel
Inscription (Estimé)
20
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
- Nom: Ipsen Clinical Study Enquiries
- Numéro de téléphone: See e-mail
- E-mail: clinical.trials@ipsen.com
Lieux d'étude
-
-
-
Beijing, Chine
- Actif, ne recrute pas
- Capital Children's Medical Center
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Chongqing, Chine
- Actif, ne recrute pas
- Chongqing Medical University - Children's Hospital
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Hunan, Chine
- Recrutement
- Hunan Children's Hospital
-
Nanjing, Chine
- Actif, ne recrute pas
- Nanjing Medical University-Nanjing Children's Hospital
-
Shanghai, Chine
- Recrutement
- Children's Hospital of Fudan University Endocrinology and Metabolism
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Xiamen, Chine
- Actif, ne recrute pas
- Xiamen Children's Hospital
-
-
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 Progressive Familial Intrahepatic Cholestasis (PFIC) (all types) who have been prescribed odevixibat by their treating physician will be eligible.
Participants who started odevixibat treatment before the implementation of the registry may also be enrolled.
La description
Inclusion Criteria:
- Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enroll the participant in this registry) by their treating physician
- On (or starting) active odevixibat treatment (participants can remain in the registry during odevixibat treatment interruptions)
- Signed informed consent and assent, as appropriate
Exclusion Criteria:
- Currently participating in a clinical trial with odevixibat
- Currently participating in any interventional clinical trial for PFIC
- Have any contraindication to odevixibat as per the approved label in China
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), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
|
An AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
|
|
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), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
|
SAEs are collected as part of safety reporting and include events meeting seriousness criteria (e.g., death, life-threatening, hospitalization, etc.) as defined in the protocol
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Event-free survival (EFS)
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection
|
EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death.
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection
|
|
Surgical biliary diversion-free survival
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
|
Liver transplant-free survival
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
|
Overall survival
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
Defined as time from the start of odevixibat treatment to death.
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
|
Pruritus improvement
Délai: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Pruritus improvement described at each patient visit using a (semi-)objective scoring scale to assess level of pruritus from the start of the odevixibat treatment.
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
|
Change from baseline in serum bile acid
Délai: From baseline and up to end of data collection (approximately 5 years of data collection
|
Change from baseline assessed by measuring serum bile acid levels at each patient visit.
|
From baseline 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
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)
16 avril 2026
Achèvement primaire (Estimé)
30 avril 2031
Achèvement de l'étude (Estimé)
30 avril 2031
Dates d'inscription aux études
Première soumission
8 mai 2026
Première soumission répondant aux critères de contrôle qualité
14 mai 2026
Première publication (Réel)
15 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-60240-032
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
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