Clinical Characterization of Patients With Primary Biliary Cirrhosis Treated With Seladelpar in the Real-life Setting
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 100
- 试验地点
- 21
- 主要终点
- biochemical response according to POISE criteria
研究概览
简要总结
The seladelpar registry will collect real-world data of patients with PBC diagnosis treated with seladelpar in the real-life scenario in Germany and Switzerland.
详细描述
The seladelpar registry will offer data acquisition at weeks 0 - 4 - 8 - 12 - 24 - 36 - 48 of seladelpar treatment. Patients can be recruited either retro- or prospectively. The seladelpar registry will be an interim project until the start of the PBC 2.0 registry. Patients within the seladelpar registry will be characterized in detail with drug specific aspects, before it will be offered to transfer data for a long-term follow-up in the German PBC 2.0 registry if patients agree to this procedure in an upcoming informed consent process once the PBC 2.0 registry will be initiated.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 18 years
- •Diagnosis of PBC according to EASL criteria
- •Treatment with seladelpar
- •Written informed consent
排除标准
- •current or previous participation in a phase I to IV interventional clinical trial for seladelpar treatment of PBC
- •Pregnancy and breastfeeding
研究组 & 干预措施
patients with primary biliary cirrhosis treated with seladelpar
Patients with primary biliary cirrhosis treated with seladelpar will be included into the SEL registry. No interventions. Routine data is collected. The documentation of the routine data is carried out alongside with guideline recommended treatment intervals of the patients.
结局指标
主要结局
biochemical response according to POISE criteria
时间窗: start of seladelpar therapy (week 0) until week 48 after start of seladelpar
The endpoint "POISE criteria" is reached if at the 48 week visit, ALP \< 1.67 x ULN and at least 15% lower than week 0 value from the start of seladelpar
次要结局
- alternative definitions of response at the 48 week visit for patients with ALP levels > 1.5 or > 1.0 x ULN or bilirubin levels > 1.0 or > 0.6 x ULN at week 0 of seladelpar therapy(start of seladelpar therapy (week 0) until week 48 after start of seladelpar)
- Improvement in Vibration Controlled Transient Elastography (VCTE)(start of seladelpar therapy (week 0) until week 48 after start of seladelpar)
- indication for seladelpar(start of seladelpar therapy (week 0) until week 48 after start of seladelpar)
- AEs and SAEs will be classified using the Medical Dictionary for Regulatory Activities (MedDRA)(start of seladelpar therapy (week 0) until week 48 after start of seladelpar)
- concomitant diseases(start of seladelpar therapy (week 0) until week 48 after start of seladelpar)
- concomitant medication classes(start of seladelpar therapy (week 0) until week 48 after start of seladelpar)
- problems with pruritus(start of seladelpar therapy (week 0) until week 48 after start of seladelpar)
- questionnaire(start of seladelpar therapy (week 0) until week 48 after start of seladelpar (Questionnaires can only be collected prospectively. The number of questionnaires depends on the time of patient recruitment.))
研究者
Johannes Wiegand
Prof. Dr.
University of Leipzig