Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Ipsen
- 入组人数
- 30
- 试验地点
- 11
- 主要终点
- Percentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation.
研究概览
简要总结
This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives.
Odevixibat is a medicine that helps patients with ALGS, a rare disease that harms their liver and causes itching.
The main aim of this study is to observe the long-term, everyday effectiveness and safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with ALGS.
- •On (or starting) active odevixibat treatment.
- •Signed informed consent and assent, as appropriate. Consent/assent from the participant or legal representative should be obtained, as appropriate, before any study data collection is conducted. Participants who turn 18 years of age (or legal age per country) while participating in the study will be required to provide consent for themselves.
排除标准
- •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.
- •Had liver transplant before enrolment
结局指标
主要结局
Percentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation.
时间窗: From first dose to end of study (approximately 5 years data collection)
Surgical biliary diversion-free survival
时间窗: From first dose to end of study (approximately 5 years data collection)
Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
Liver transplant-free survival
时间窗: From first dose to end of study (approximately 5 years data collection)
Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
Overall survival
时间窗: From first dose to end of study (approximately 5 years data collection)
Defined as time from the start of odevixibat treatment to death.
次要结局
- Change from Baseline in Weight(From first dose to end of study (approximately 5 years data collection))
- Change from Baseline in Height(From first dose to end of study (approximately 5 years data collection))
- Percentage of participants with Adverse Events (AEs) associated with fat-soluble vitamin (FSV) deficiencies and their possible sequelae.(From signing of the Informed Consent Form (ICF) to 180 days after the last dose intake of odevixibat)
- Percentage of participants with suspected hepatotoxic Adverse events (AEs) requiring interruption of odevixibat(From signing of the ICF to 180 days after the last dose intake of odevixibat)
- Percentage of participants with bleeding AEs(From signing of the ICF to 180 days after the last dose intake of odevixibat)
- Percentage of participants with AEs(From signing of the ICF to 180 days after the last dose intake of odevixibat)