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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

2026年8月28日 更新者：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.

研究概览

地位

招聘中

条件

研究类型

观察性的

注册 (估计的)

20

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

学习地点

  • 中国
      • Beijing、中国
        • 主动，不招人
        • Capital Children's Medical Center
      • Chongqing、中国
        • 主动，不招人
        • Chongqing Medical University - Children's Hospital
      • Hunan、中国
        • 招聘中
        • Hunan Children's Hospital
      • Nanjing、中国
        • 主动，不招人
        • Nanjing Medical University-Nanjing Children's Hospital
      • Shanghai、中国
        • 招聘中
        • Children's Hospital of Fudan University Endocrinology and Metabolism
      • Xiamen、中国
        • 主动，不招人
        • Xiamen Children's Hospital

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 孩子
  • 成人
  • 年长者

接受健康志愿者

不

取样方法

非概率样本

研究人群

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.

描述

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

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Percentage of participants experiencing adverse events (AEs)
大体时间：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)
大体时间：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.

次要结果测量

结果测量
措施说明
大体时间
Event-free survival (EFS)
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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
大体时间：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

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Ipsen

调查人员

  • 研究主任：Ipsen Medical Director、Ipsen

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2026年4月16日

初级完成 (估计的)

2031年4月30日

研究完成 (估计的)

2031年4月30日

研究注册日期

首次提交

2026年5月8日

首先提交符合 QC 标准的

2026年5月14日

首次发布 (实际的)

2026年5月15日

研究记录更新

最后更新发布 (实际的)

2026年8月31日

上次提交的符合 QC 标准的更新

2026年8月28日

最后验证

2026年8月1日

更多信息

与本研究相关的术语

其他相关的 MeSH 术语

其他研究编号

  • CLIN-60240-032

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

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.

IPD 共享时间框架

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

IPD 共享访问标准

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

药物和器械信息、研究文件

研究美国 FDA 监管的药品

不

研究美国 FDA 监管的设备产品

不

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