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 de agosto de 2026 atualizado por: 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.
Visão geral do estudo
Status
Recrutamento
Tipo de estudo
Observacional
Inscrição (Estimado)
20
Contactos e Locais
Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.
Contato de estudo
- Nome: Ipsen Clinical Study Enquiries
- Número de telefone: See e-mail
- E-mail: clinical.trials@ipsen.com
Locais de estudo
-
-
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Beijing, China
- Ativo, não recrutando
- Capital Children's Medical Center
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Chongqing, China
- Ativo, não recrutando
- Chongqing Medical University - Children's Hospital
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Hunan, China
- Recrutamento
- Hunan Children's Hospital
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Nanjing, China
- Ativo, não recrutando
- Nanjing Medical University-Nanjing Children's Hospital
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Shanghai, China
- Recrutamento
- Children's Hospital of Fudan University Endocrinology and Metabolism
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Xiamen, China
- Ativo, não recrutando
- Xiamen Children's Hospital
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-
Critérios de participação
Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.
Critérios de elegibilidade
Idades elegíveis para estudo
- Filho
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Não
Método de amostragem
Amostra Não Probabilística
População do estudo
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.
Descrição
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
Plano de estudo
Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.
Como o estudo é projetado?
Detalhes do projeto
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Percentage of participants experiencing adverse events (AEs)
Prazo: 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)
Prazo: 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
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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.
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Event-free survival (EFS)
Prazo: 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
Prazo: 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)
|
|
Liver transplant-free survival
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
|
Colaboradores e Investigadores
É aqui que você encontrará pessoas e organizações envolvidas com este estudo.
Patrocinador
Investigadores
- Diretor de estudo: Ipsen Medical Director, Ipsen
Datas de registro do estudo
Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.
Datas Principais do Estudo
Início do estudo (Real)
16 de abril de 2026
Conclusão Primária (Estimado)
30 de abril de 2031
Conclusão do estudo (Estimado)
30 de abril de 2031
Datas de inscrição no estudo
Enviado pela primeira vez
8 de maio de 2026
Enviado pela primeira vez que atendeu aos critérios de CQ
14 de maio de 2026
Primeira postagem (Real)
15 de maio de 2026
Atualizações de registro de estudo
Última Atualização Postada (Real)
31 de agosto de 2026
Última atualização enviada que atendeu aos critérios de controle de qualidade
28 de agosto de 2026
Última verificação
1 de agosto de 2026
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- CLIN-60240-032
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
SIM
Descrição do plano 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.
Prazo de Compartilhamento de 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
Critérios de acesso de compartilhamento 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/).
Informações sobre medicamentos e dispositivos, documentos de estudo
Estuda um medicamento regulamentado pela FDA dos EUA
Não
Estuda um produto de dispositivo regulamentado pela FDA dos EUA
Não
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