A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease
研究概览
详细说明
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).
研究类型
注册 (实际的)
联系人和位置
学习地点
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Alabama
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Huntsville、Alabama、美国、35805
- Nephrology Consultants, LLC
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
取样方法
研究人群
描述
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
学习计划
研究是如何设计的?
设计细节
研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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General clinical condition
大体时间:Approximately every 3 months
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General clinical condition
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Approximately every 3 months
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Proportion of patients with episodes or exacerbations of medical events
大体时间:Approximately every 3 months
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Proportion of patients with episodes or exacerbations of medical events
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Approximately every 3 months
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Proportion of patients with infections As applicable, infections (particularly meningococcal and other encapsulated bacteria)
大体时间:Approximately every 3 months
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Proportion of patients with infections
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Approximately every 3 months
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Proportion of patients with kidney disease progression
大体时间:Approximately every 3 months
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Proportion of patients with kidney disease progression
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Approximately every 3 months
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合作者和调查者
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.