A Post-marketing Surveillance of Fabhalta® (Iptacopan) in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) or C3 Glomerulopathy (C3G)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 21
- 试验地点
- 3
- 主要终点
- Incidence Rate of Adverse Events (AE), Serious Adverse Events (SAE), and Adverse Drug Reactions (ADR)
研究概览
简要总结
This is a post-marketing surveillance study conducted as part of the Risk Management Plan (RMP) for South Korea, to evaluate the safety and effectiveness of iptacopan in real-world clinical settings for the treatment of either PNH or C3G in Korean patients. Prospective data will be collected from patient medical records to address the objectives for all eligible populations.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged 18 years or older who have been diagnosed with PNH or C3G.
- •Patients who have received vaccination in accordance with the approved Korean labeling prior to initiating treatment with iptacopan.
- •Patients who are being treated or will be treated with iptacopan in accordance with the approved Korean labeling.
- •Patients who have voluntarily provided consent for study participation (written informed consent).
排除标准
- •Patients who fall under the contraindications for iptacopan administration according to approved Korean labeling.
- •Patients for whom iptacopan administration is deemed inappropriate based on the investigator's judgment.
研究组 & 干预措施
PNH Cohort
Patients in South Korea who receive at least one dose of iptacopan for the treatment of PNH.
C3G Cohort
Patients in South Korea who receive at least one dose of iptacopan for the treatment of C3G.
结局指标
主要结局
Incidence Rate of Adverse Events (AE), Serious Adverse Events (SAE), and Adverse Drug Reactions (ADR)
时间窗: Up to 2 years
Incidence rate of all AE/ADR, SAE/serious adverse drug reactions (SADR), unexpected AE/ADR, and unexpected SAE/SADR that occur after the administration of iptacopan.
次要结局
- Estimated Glomerular Filtration Rate (eGFR)(Baseline and Week 24)
- Hemoglobin Levels(Baseline and Week 24)
- Lactate Dehydrogenase (LDH) levels(Baseline and Week 24)
- White Blood Cell Count (WBC)(Baseline and Week 24)
- Red Blood Cell Count (RBC)(Baseline and Week 24)
- Hematocrit Levels(Baseline and Week 24)
- Platelet Count(Baseline and Week 24)
- Reticulocyte Count(Baseline and Week 24)
- Ferritin Levels(Baseline and Week 24)
- Total Bilirubin Levels(Baseline and Week 24)
- Blood Urea Nitrogen (BUN) Levels(Baseline and Week 24)
- Creatinine Levels(Baseline and Week 24)
- Log-transformed Ratio to Baseline in Urine Protein Creatinine Ratio (UPCR) [Random Spot Urine Test](Week 24)
- Change From Baseline in Serum Creatinine(Baseline and Week 24)
- Change From Baseline in eGFR(Baseline and Week 24)
- Overall Improvement Assessed by Investigator(Week 24)