NCT06931691招募中不适用
A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan on Disease Management, Treatment-Related Outcomes and Healthcare Resource Utilization for Adult Patients With Paroxysmal Nocturnal Hemoglobinuria in China
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 80
- 试验地点
- 14
- 主要终点
- Change from baseline in hemoglobin (Hb) levels at designated time points
研究概览
简要总结
The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients.
Based on these backgrounds, we plan to conduct a real-world study of iptacopan to further evaluate its impact on treatment-related outcomes, disease management, and healthcare resource utilization in Chinese patients with PNH.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •For Cohort 1,
- •Patient who meets all the following criteria can be included in this study:
- •Age ≥ 18 years at the time of signing the ICF;
- •Patient with a documented diagnosis of PNH;
- •Patient who has never received complement inhibitor therapy;
- •Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
- •Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation; If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible.
- •Patient who has signed the ICF.
- •For Cohort 2,
- •Patient who meets all the following criteria can be included in this study:
- •Age ≥ 18 years at the time of signing the ICF;
- •Patient with a documented diagnosis of PNH; Patients who have been receiving stable treatment with C5 complement inhibitors for at least three months prior to enrollment;
- •Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
- •Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation;
- •If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible.
- •Patient who has signed the ICF.
排除标准
- •For Cohort 1 and Cohort 2, patients who meet any of the following criteria will meet the exclusion criteria for this study:
- •Participating in an interventional PNH clinical study;
- •Have an active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to first dose;
- •Documented with a history of recurrent invasive infections, e.g. active systemic bacterial, viral or fungal infection within 14 days prior to first dose;
- •Documented with a history of HIV infection;
- •Women who are pregnant or breastfeeding or intending to conceive during the study period;
- •Existence of bone marrow failure (reticulocytes < 100 × 109/L, platelets < 30 × 109/L, and neutrophils < 0.5 × 109/L) determined by the investigator;
- •Other conditions that are not suitable for participating in the study, in the judgment of the investigator.
研究组 & 干预措施
Cohort1
PNH patients with a confirmed diagnosis of PNH in the medical record and who had never received complement inhibitor therapy (complement inhibitor naive patients)
干预措施: LNP023 (Drug)
Cohort 2
PNH patients with a confirmed diagnosis of PNH in the medical record who had been treated with a stable dose of a C5 complement inhibitor (e.g., eculizumab) for at least 3 months (C5i-treated patients; this cohort will be only start enrollment after the indication for iptacopan is to be approved in China).
干预措施: LNP023 (Drug)
结局指标
主要结局
Change from baseline in hemoglobin (Hb) levels at designated time points
时间窗: Baseline, 12 months
Assessment of the Hematological Response to iptacopan.
次要结局
- Number of participants having Hb normalization (Hb level ≥ 12 g/dL) after iptacopan initiation.(12 months)
- Duration of Hb level ≥ 12 g/dL within a 12-month(12 months)
- Change from baseline in LDH Levels after iptacopan initiation(Baseline, 12 Months)
- Number of participants with LDH levels ≤ 1.5 x ULN before vs. after iptacopan initiation(12 months)
- Number of participants having LDH normalization before and after iptacopan initiation(12 monhts)
- Duration of LDH level ≤ 1.5 x ULN within 12 months(12 months)
- Change from baseline in ARC Levels after iptacopan initiation(Baseline, 12 monhts)
- Number of participants having ARC normalization before and after iptacopan initiation(Baseline, 12 months)
- Change from baseline in bilirubin after iptacopan initiation(Baseline, 12 months)
- Number of participants with hepatosplenomegaly before and after iptacopan initiation(Baseline, 12 months)
- Number of participants with a positive coomb's test after iptacopan initiation(12 months)
- Number of participants with PNH related signs or symptoms before and after iptacopan treatment(Baseline, 12 months)
- Change from baseline in PNH clone value(Baseline, 12 months)
- Rate, reasons (complement activation conditions (CAC), missed doses, etc.), duration, and interventions for breakthrough hemolysis (BTH)(12 months)
- Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale after iptacopan initiation(Baseline, 12 months)
- Work Productivity and Activity Impairment Questionnaire-Specific Health Problems (WPAI-SHP) at each visit, change from baseline in WPAI-SHP after iptacopan initiation(Baseline, 12 months)
- Adverse events (AEs) and Serious adverse events (SAEs) occurring during treatment with iptacopan(12 monhts)
- Treatment regimen for PNH 12 months before iptacopan treatment; reasons of discontinuation of previous complement inhibitor;(Baseline)
- Use of concomitant medications (e.g., corticosteroids, androgen, immunosuppressants, anti-coagulants, etc.) for PNH and PNH-related complications (e.g. thrombosis, renal failure, etc.) before and after iptacopan treatment;(Baseline, 12 months)
- Duration of iptacopan treatment, reasons for discontinuation, switching to other treatments, proportion of patients who switch from iptacopan to other treatments;(12 monhts)
- Number of participants with blood transfusion before and after iptacopan treatment, duration and number of units of blood transfusion due to BTH(Baseline, 12 months)
- Number of participants hospitalized, outpatient, emergency room and intensive care unit (ICU) visits related to PNH(12 monhts)
- Length of inpatient stay related to PNH(12 months)
研究者
研究点 (14)
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