Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the Non-interventional IPIG PNH Registry
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- Number of patients with infections caused by encapsulated bacteria
研究概览
简要总结
This is an observational single-arm descriptive cohort study based on the secondary use of data collected on iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry.
详细描述
This multinational, non-interventional, descriptive single-arm cohort study is based on secondary analysis of data collected within the iptacopan silo of the IPIG PNH Registry (data on iptacopan-treated patients made available to Novartis). This is a non-interventional study utilizing secondary data and is considered a "registry-based study." The IPIG PNH Registry (CT.gov NCT06524726), the parent registry, includes a dedicated drug silo to collect data from patients using iptacopan in routine care.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent to participate in the IPIG PNH Registry
- •PNH confirmed by flow cytometry
- •Incident users of iptacopan
- •Aged at least 18 years at the iptacopan initiation
排除标准
- •Participation in an interventional clinical trial
研究组 & 干预措施
Iptacopan
Adult patients with PNH treated with iptacopan in routine care.
干预措施: Iptacopan (Drug)
结局指标
主要结局
Number of patients with infections caused by encapsulated bacteria
时间窗: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
Number of infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria
时间窗: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
Cumulative incidence of infections (event probability as a function of time), caused by encapsulated bacteria
时间窗: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
Number of patients with infections events per 100 participants -years (incidence rates) caused by encapsulated bacteria
时间窗: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
次要结局
- Number of patients with serious infections caused by encapsulated bacteria and all serious infection(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)
- Number of patients vaccinated against Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae at each study visit(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)
- Number of patients with serious hemolysis following discontinuation of iptacopan(From the iptacopan discontinuation up to 14 days)
- Number of patients who became pregnant during treatment with iptacopan, exposure characteristics (e.g. trimester of exposure) and birth outcomes(From the Last Menstrual Period to pregnancy outcome (in case of live birth, up to 12 months post delivery))
- Cumulative incidence of serious infections, caused by encapsulated bacteria and all serious infection (event probability as a function of time)(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)
- Number of patients with serious infections events per 100 patients -years (incidence rates) caused by encapsulated bacteria and all serious infection(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)
- Number of serious infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria and all serious infection(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)
- Number of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia episodes per 100 patients -years (occurrence rates)(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)
- Number of patients with death due to any cause(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)
- Cumulative incidence of death due to any cause (event probability as a function of time)(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)
- Number of patients with death due to any cause events per 100 patients -years (incidence rates)(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)
- Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, Major adverse vascular events (MAVEs), serious adverse events (SAEs), hyperlipidemia and thrombocytopenia(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)
- Cumulative incidence of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia (event probability as a function of time)(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)
- Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia events per 100 patients -years (incidence rates)(From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.)