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
14 maggio 2026 aggiornato da: 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.
Panoramica dello studio
Stato
Reclutamento
Condizioni
Tipo di studio
Osservativo
Iscrizione (Stimato)
20
Contatti e Sedi
Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.
Contatto studio
- Nome: Ipsen Clinical Study Enquiries
- Numero di telefono: See e-mail
- Email: clinical.trials@ipsen.com
Luoghi di studio
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Shanghai, Cina
- Reclutamento
- Children's Hospital of Fudan University Endocrinology and Metabolism
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Criteri di partecipazione
I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.
Criteri di ammissibilità
Età idonea allo studio
- Bambino
- Adulto
- Adulto più anziano
Accetta volontari sani
No
Metodo di campionamento
Campione non probabilistico
Popolazione di studio
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.
Descrizione
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
Piano di studio
Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.
Come è strutturato lo studio?
Dettagli di progettazione
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
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Percentage of participants experiencing adverse events (AEs)
Lasso di tempo: 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.
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An AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment
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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.
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Percentage of participants experiencing serious adverse events (SAEs)
Lasso di tempo: 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.
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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
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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.
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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Event-free survival (EFS)
Lasso di tempo: From first ICF signature and up to end of data collection (approximately 5 years of data collection
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EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection
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Surgical biliary diversion-free survival
Lasso di tempo: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Liver transplant-free survival
Lasso di tempo: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Overall survival
Lasso di tempo: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Defined as time from the start of odevixibat treatment to death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Pruritus improvement
Lasso di tempo: 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.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Change from baseline in serum bile acid
Lasso di tempo: From baseline and up to end of data collection (approximately 5 years of data collection
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Change from baseline assessed by measuring serum bile acid levels at each patient visit.
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From baseline and up to end of data collection (approximately 5 years of data collection
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Collaboratori e investigatori
Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.
Sponsor
Investigatori
- Direttore dello studio: Ipsen Medical Director, Ipsen
Studiare le date dei record
Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.
Studia le date principali
Inizio studio (Effettivo)
16 aprile 2026
Completamento primario (Stimato)
30 aprile 2031
Completamento dello studio (Stimato)
30 aprile 2031
Date di iscrizione allo studio
Primo inviato
8 maggio 2026
Primo inviato che soddisfa i criteri di controllo qualità
14 maggio 2026
Primo Inserito (Effettivo)
15 maggio 2026
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
15 maggio 2026
Ultimo aggiornamento inviato che soddisfa i criteri QC
14 maggio 2026
Ultimo verificato
1 maggio 2026
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- CLIN-60240-032
Piano per i dati dei singoli partecipanti (IPD)
Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?
SÌ
Descrizione del piano 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.
Periodo di condivisione 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
Criteri di accesso alla condivisione 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/).
Informazioni su farmaci e dispositivi, documenti di studio
Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti
No
Studia un dispositivo regolamentato dalla FDA degli Stati Uniti
No
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .