A Study to Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of International PNH Interest Group (IPIG)-Registry Data
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry. The primary objectives seek to characterize the long-term safety profile of danicopan as add-on therapy to ravulizumab/eculizumab in participants with PNH.
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
- •Adult participants aged ≥ 18 years at treatment initiation.
- •Initiated treatment with Ultomiris, Soliris, and/or danicopan on or after IPIG or Alexion International PNH Registry enrollment.
排除标准
- •Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and Ultomiris and/or Soliris.
研究组 & 干预措施
Cohort 1
Participants ever-treated with danicopan as add-on therapy to ravulizumab/eculizumab on or after IPIG PNH Registry enrollment.
干预措施: Danicopan (Drug)
Cohort 2
Participants initiating treatment with Ultomiris or Soliris on or after IPIG or Alexion International PNH Registry enrollment and without any danicopan treatment experience during follow-up.
干预措施: Soliris/Ultomiris (Drug)
结局指标
主要结局
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years
Event Rate of Meningococcal Infections
时间窗: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years
Event Rate of Serious Infections
时间窗: Up to approximately 5 years
Event Rate of Malignancies and Hematologic Abnormalities
时间窗: Up to approximately 5 years
次要结局
- Number of Participants with PNH symptoms at Diagnosis(Baseline (Day 1))
- Number of Participants with AEs and SAEs in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding(Up to approximately 5 years)
- Number of Infant Health Abnormalities in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding(Up to 12 months)
- Number of Participants with a History Bone Marrow Transplant(Baseline (Day 1))
- Number of Participants with a history of Major Adverse Vascular Events, including Thrombosis(Baseline (Day 1))
- Number of Participants with Ongoing Severe Hepatic Impairment as Defined by Child-Pugh Class C at Enrollment(Baseline (Day 1))
- Number of Participants Who Discontinue Danicopan Treatment(Up to approximately 5 years)