A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- General clinical condition
研究概览
简要总结
This study will follow participants with a disease which is associated with an absence or deficiency in Complement Factor I (CFI) as confirmed in the CFI-001 screening study.
详细描述
This study will follow participants with a disease which is associated with an absence or deficiency in Complement Factor I (CFI) as confirmed in the CFI-001 screening study.
The population will include male and female participants ≥6 months of age (pediatric participants enrolled as permitted by local Institutional Review Board/Independent Ethics Committee). This includes patients with recurrent bacterial infections (eg, meningitis, sepsis, pneumonia, endocarditis, otitis) and patients with autoimmune and immune complex-mediated diseases (eg, glomerulonephritis, systemic lupus erythematosus, leukocytoclastic vasculitis, Bickerstaff encephalitis, meningoencephalitis).
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female, age 6 months or older
- •Affirmation of participant's informed consent or legally authorized representative (LAR)'s willingness to provide informed consent with signature confirmation before any study-related activities. (Study-related activities are any procedures that would not have been performed during normal clinical management of the participant.) The participant (if a minor) must also be willing to give written informed assent (if able) if the minor is within the age groups 7 to 11 years old and 12 to 17 years old.
- •Confirmed CFI deficiency during the screening study, CFI-001, with a disease that is associated with an absence or deficiency in CFI
- •Stated willingness of the participant to comply with all study procedures (including multiple blood draws) and availability for the duration of the study
- •Stated willingness of the participant (or LAR) to allow access to his/her medical records with the purpose of assessing disease status and progression during his/her participation in the study
排除标准
- •Participation in an interventional clinical study within the previous 30 days prior to screening or within ≤5 half-lives of the investigational drug, whichever is longer
- •Having a major concurrent non-CFI-related disease that prevents the assessment of the natural course of the CFI deficiency disease
- •Having a medical, psychosocial, or familial issues that might prevent full participation and cooperation with the procedures and requirements of the clinical study as determined by the potential participant/guardian and physician investigator
结局指标
主要结局
General clinical condition
时间窗: Approximately every 3 months
General clinical condition
Proportion of patients with kidney disease progression
时间窗: Approximately every 3 months
Proportion of patients with kidney disease progression
Proportion of patients with infections As applicable, infections (particularly meningococcal and other encapsulated bacteria)
时间窗: Approximately every 3 months
Proportion of patients with infections
Proportion of patients with episodes or exacerbations of medical events
时间窗: Approximately every 3 months
Proportion of patients with episodes or exacerbations of medical events
次要结局
未报告次要终点