Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 6,061
- 试验地点
- 1
- 主要终点
- Evaluate safety data specific to the use of Soliris and Ultomiris
研究概览
简要总结
This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
详细描述
Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients of any age, including minors, with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment. Patients who are minors must have parent/legal guardian consent and must be willing and able to give assent, if applicable as determined by the Ethics Committees/Institutional Review Boards. Upon attaining adulthood, these patients must be re-consented.
- •Ability to comprehend and sign consent to have data entered in the PNH Registry.
排除标准
- •Inability or unwillingness to sign informed consent.
- •Patients currently enrolled in an interventional clinical trial for treatment of PNH cannot be enrolled in the Registry at the same time.
结局指标
主要结局
Evaluate safety data specific to the use of Soliris and Ultomiris
时间窗: Ongoing (up to 13 years)
Primary analyses will assess safety endpoints, including occurrence and time to first event for the following: meningococcal infections, infections with serious outcomes, formation of human anti-drug antibodies (ADA) to Soliris and Ultomiris, malignancy, thrombotic events, pulmonary hypertension, impaired renal function, impaired hepatic function, hemolysis, pregnancies, lactation, infusion reactions, bone marrow transplant, serious adverse events, and mortality.
次要结局
- Collect data to characterize the progression of PNH as well as clinical outcomes, mortality and morbidity in Soliris or Ultomiris and non-Soliris or non- Ultomiris treated patients(Ongoing (up to 13 years))