Danicopan Regulatory Post-Marketing Study in Korea
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- AstraZeneca
- 入组人数
- 27
- 试验地点
- 4
- 主要终点
- Safety (adverse events (AEs), serious AEs (SAEs), adverse drug reactions (ADRs), serious ADRs (SADRs), unexpected AEs/ADRs), AESI
研究概览
简要总结
As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients who are treated with Danicopan as an add-on to ravulizumab or eculizumab in normal clinical practice settings. This study is designed to assess the known safety profile or identify previously unsuspected adverse reactions and to evaluate the effectiveness of Danicopan under conditions of routine daily medical practice in Korea.
详细描述
The objectives of this study are to assess the safety and effectiveness of Danicopan in a real world setting in patients who are prescribed with the study drug under the approved indication in Korea.
Primary objective(s) To assess the safety of Danicopan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab)in patients with PNH in Korea.
Secondary objective(s) To assess effectiveness of Danicopan as add- on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patients with PNH at 12 and 24 weeks.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients eligible for and treated with Danicpan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patient with PNH in Korea
- •Provision of a signed and dated written informed consent by the patient or their legally acceptable representative
排除标准
- •Participation in any concurrent interventional trials during the period of study drug treatment
- •Other off-label indications according to the approved label in Korea
结局指标
主要结局
Safety (adverse events (AEs), serious AEs (SAEs), adverse drug reactions (ADRs), serious ADRs (SADRs), unexpected AEs/ADRs), AESI
时间窗: Patient data will be gathered for up to 24 weeks from the first dose of the study drug.
To assess the safety of Danicopan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab)in patients with PNH in Korea.
Safety (adverse events (AEs), serious AEs (SAEs), adverse drug reactions (ADRs), serious ADRs (SADRs), unexpected AEs/ADRs), AESI
时间窗: Patient data will be gathered for up to 12 weeks from the first dose of the study drug.
To assess the safety of Danicopan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab)in patients with PNH in Korea.
次要结局
- Change from baseline in hemoglobin (Hgb) at Week 12 and 24(Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24).)
- Change from baseline in absolute reticulocyte count (ARC) at Week 12 and 24(Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24))
- Change from baseline in FACIT Fatigue scores at Week 12 and 24(Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24))
- Proportion of patients with transfusion avoidance* at Week 12 and 24 (* Transfusion Avoidance: Defined as remaining free from red blood cell transfusions)(Effectiveness variables will be assessed at Week 12 and 24 or end of treatment (if treatment is discontinued before Week 24))
- Change from baseline in hemoglobin (Hgb) at Week 12(Effectiveness variables will be assessed at Week 12 or end of treatment (if treatment is discontinued before Week 12).)
- Change from baseline in absolute reticulocyte count (ARC) at Week 12(Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12))
- Change from baseline in FACIT Fatigue scores at Week 12(Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12))
- Proportion of patients with transfusion avoidance* at Week 12(* Transfusion Avoidance: Defined as remaining free from red blood cell transfusions)(Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12))