NCT06447168招募中不适用
Prospective Non-interventional, Phase IV Multicentre Study to Assess the Effectiveness, Safety and Tolerability of Elafibranor 80 mg/Day in Participants With Primary Biliary Cholangitis Receiving Treatment in a Real-world Setting.
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Ipsen
- 入组人数
- 424
- 试验地点
- 136
- 主要终点
- Percentage of participants with response to treatment
研究概览
简要总结
This study will collect information from participants with Primary Biliary Cholangitis (PBC) as they use the drug elafibranor in real world setting.
PBC is a progressive rare liver disease in which tubes in the liver called bile ducts are damaged.
The liver damage in PBC may lead to scarring (cirrhosis). PBC may also be associated with multiple symptoms including pruritus (itching) and fatigue. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done.
In this study the main aim is to observe the effectiveness, safety and tolerability of elafibranor in participants with PBC who are receiving treatment in real world setting. The total study duration for each participants will be 60 months (approximately 5 years).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant has provided written informed consent and agrees to comply with the study protocol.
- •Participant with PBC diagnosis.
- •Participant for whom the treating physician has decided to start or participants who are currently receiving treatment with commercialized elafibranor.
- •If a participant has a caregiver who agrees to complete the caregiver questionnaires, an informed consent should be collected from the caregiver before any data is collected.
排除标准
- •Participant is currently participating or, plans to participate in an investigational drug study or medical device study containing active substance.
- •Participant with known hypersensitivity to the product or to any of its excipients.
- •Participant with mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.
结局指标
主要结局
Percentage of participants with response to treatment
时间窗: At month 6
Defined as alkaline phosphatase (ALP) \<1.67 x upper limit of normal (ULN) and total bilirubin (TB) ≤ULN and ALP decrease ≥15% from baseline.
次要结局
- Percentage of participants with normalization of ALP levels(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Percentage of participants with response to treatment(Up to 60 months following enrollment, based on routine physician follow-up visits)
- Change from baseline in liver function parameters: Serum levels of alanine aminotransferase (ALT)(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in liver function parameters: Serum levels of Aspartate aminotransferase (AST)(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in liver function parameters: Serum levels of Gamma-glutamyl transferase (GGT)(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in liver function parameters: Serum levels of TB(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in liver function parameters: Conjugated (direct) bilirubin(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in liver function parameters: Serum levels of creatinine(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in liver function parameters: Serum levels of albumin(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in pruritus based on PBC Itch score(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in fatigue based on functional assessment of chronic illness therapy-fatigue (FACIT-Fatigue) scale(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in sleep based on Pittsburgh sleep quality index (PSQI)(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in Quality Of Life (QoL) based on PBC-40 questionnaire(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in QoL based on 5-Dimensional Itch scale (5-D Itch, also known as 5-D pruritus scale)(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Change from baseline in liver stiffness(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Event free survival(From baseline to up to 60 months)
- Percentage of participants experiencing Adverse Events (AEs), Adverse Events of Special Interests (AESIs) and special situations (SS).(From baseline to up to 60 months)
- Participant's satisfaction on treatment(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Participant's adherence to treatment(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Percentage of participants with clinically significant changes in laboratory parameters(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Percentage of participants with clinically significant changes in physical examination(Up to 60 months following enrollment, based on routine physician follow-up visit)
- Percentage of participants developing clinically significant changes in vital signs(Up to 60 months following enrollment, based on routine physician follow-up visit)
研究者
研究点 (136)
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