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A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment

2026年10月20日 更新者：Ipsen

Prospective Non-Interventional Study Evaluating the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS)

This study will collect information from patients with ALGS who are using odevixibat in their daily lives. Odevixibat is a medication that helps patients with ALGS, a rare disease that affects the liver and causes itching.

The main aim of this study is to observe the long-term, everyday safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.

研究概览

地位

招聘中

条件

研究类型

观察性的

注册 (估计的)

30

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

  • 意大利
      • Florence、意大利
        • 招聘中
        • Azienda Ospedaliero Universitaria Meyer
      • Naples、意大利
        • 招聘中
        • Azienda Ospedaliera Di Rilievo Nazionale Santobono Pausilipon
      • Padova、意大利
        • 尚未招聘
        • University Hospital Of Padova
      • Roma、意大利
        • 尚未招聘
        • IRCCS Ospedale Pediatrico Bambino Gesu
      • Torino、意大利
        • 尚未招聘
        • A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita
      • Udine、意大利
        • 尚未招聘
        • Ospedale Universitario Di Udine
  • 法国
      • Bron、法国
        • 招聘中
        • Hospices Civils de Lyon - Hopital Femme Mere Enfant
      • Dijon、法国
        • 尚未招聘
        • CHU Dijon Bourgogne
      • Le Kremlin-Bicêtre、法国
        • 尚未招聘
        • APHP - Hopital Bicetre - Paris Sud
      • Marseille、法国
        • 尚未招聘
        • APHM - CHU Timone
      • Montpellier、法国
        • 尚未招聘
        • CHU de Montpellier - Hopital Saint Eloi
      • Paris、法国
        • 尚未招聘
        • APHP - Hopital Necker-Enfants Malades
      • Toulouse、法国
        • 尚未招聘
        • CHU de Toulouse - Hopital Paule de Viguier
      • Villejuif、法国
        • 尚未招聘
        • APHP - Centre Hepato-Biliaire Hospital Paul Brousse

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

6个月 及以上 (孩子、成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

取样方法

非概率样本

研究人群

Participants with Alagille syndrome (ALGS) who have been prescribed odevixibat by their treating physician will be enrolled into the study. Participants who started odevixibat treatment before study implementation may also be enrolled.

描述

Inclusion Criteria:

  • Diagnosed with ALGS.
  • On (or starting) active odevixibat treatment.
  • Aged 6 months or older at the time of consent.

Exclusion Criteria:

  • Currently participating in a clinical trial with odevixibat.
  • Currently participating in any interventional clinical trial for ALGS.
  • Have any contraindication to odevixibat as per the locally approved label.

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

  • 观测模型：OTHER
  • 时间观点：PROSPECTIVE

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)

An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment.
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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)

An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment.
From first ICF signature and up to end of data collection (approximately 5 years of data collection)

次要结果测量

结果测量
措施说明
大体时间
Percentage of participants with severe diarrhoea events
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with bloody diarrhoea events
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants experiencing diarrhoea events with concurrent dehydration
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants experiencing diarrhoea events treated with oral or intravenous rehydration
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in fat-soluble vitamin (FSV) levels
大体时间：From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with fat-soluble vitamin deficiency
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with suspected hepatotoxicity requiring interruption of odevixibat treatment
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with clinical manifestations related to hepatotoxicity
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in alanine aminotransferase (ALT)
大体时间：From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in aspartate aminotransferase (AST)
大体时间：From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in gamma-glutamyl transferase (GGT)
大体时间：From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in blood bilirubin
大体时间：From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in international normalized ratio (INR)
大体时间：From baseline and up to end of data collection (approximately 5 years of data collection)
From baseline and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to diarrhoea
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to hepatotoxicity
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to fat-soluble vitamin deficiency
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with treatment discontinuations due to diarrhoea
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection).
From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with treatment discontinuations due to hepatotoxicity
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection).
From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with treatment discontinuations due to fat-soluble vitamin deficiency
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection).
From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with pregnancy and maternal complications
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of foetuses, neonates, or infants with adverse effects following exposure to odevixibat during pregnancy and/or lactation
大体时间：From first documented exposure during pregnancy or lactation and up to end of data collection (approximately 5 years of data collection)
From first documented exposure during pregnancy or lactation and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with biliary diversion surgery
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with liver transplantation
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants who die from any cause
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants switching from odevixibat to maralixibat
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with clinical manifestations of fat-soluble vitamin deficiency
大体时间：From first ICF signature and up to end of data collection (approximately 5 years of data collection)

For example, bleeding, rickets, or osteopenia.
From first ICF signature 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7月10日

初级完成 (估计的)

2031年9月30日

研究完成 (估计的)

2031年9月30日

研究注册日期

首次提交

2026年5月6日

首先提交符合 QC 标准的

2026年5月6日

首次发布 (实际的)

2026年5月13日

研究记录更新

最后更新发布 (实际的)

2026年10月23日

上次提交的符合 QC 标准的更新

2026年10月20日

最后验证

2026年10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-60240-034

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

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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