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 actualizado 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.
Descripción general del estudio
Estado
Reclutamiento
Condiciones
Tipo de estudio
De observación
Inscripción (Estimado)
20
Contactos y Ubicaciones
Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.
Estudio Contacto
- Nombre: Ipsen Clinical Study Enquiries
- Número de teléfono: See e-mail
- Correo electrónico: clinical.trials@ipsen.com
Ubicaciones de estudio
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-
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Beijing, Porcelana
- Activo, no reclutando
- Capital Children's Medical Center
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Chongqing, Porcelana
- Activo, no reclutando
- Chongqing Medical University - Children's Hospital
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Hunan, Porcelana
- Reclutamiento
- Hunan Children's Hospital
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Nanjing, Porcelana
- Activo, no reclutando
- Nanjing Medical University-Nanjing Children's Hospital
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Shanghai, Porcelana
- Reclutamiento
- Children's Hospital of Fudan University Endocrinology and Metabolism
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Xiamen, Porcelana
- Activo, no reclutando
- Xiamen Children's Hospital
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-
Criterios de participación
Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.
Criterio de elegibilidad
Edades elegibles para estudiar
- Niño
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
No
Método de muestreo
Muestra no probabilística
Población de estudio
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.
Descripción
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 de estudios
Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.
¿Cómo está diseñado el estudio?
Detalles de diseño
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Percentage of participants experiencing adverse events (AEs)
Periodo de tiempo: 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)
Periodo de tiempo: 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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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Event-free survival (EFS)
Periodo de tiempo: 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.
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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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
|
Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Investigadores
- Director de estudio: Ipsen Medical Director, Ipsen
Fechas de registro del estudio
Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.
Fechas importantes del estudio
Inicio del estudio (Actual)
16 de abril de 2026
Finalización primaria (Estimado)
30 de abril de 2031
Finalización del estudio (Estimado)
30 de abril de 2031
Fechas de registro del estudio
Enviado por primera vez
8 de mayo de 2026
Primero enviado que cumplió con los criterios de control de calidad
14 de mayo de 2026
Publicado por primera vez (Actual)
15 de mayo de 2026
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
31 de agosto de 2026
Última actualización enviada que cumplió con los criterios de control de calidad
28 de agosto de 2026
Última verificación
1 de agosto de 2026
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- CLIN-60240-032
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
SÍ
Descripción del plan 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.
Marco de tiempo para compartir 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
Criterios de acceso compartido de 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/).
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
No
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
No
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