A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease

Study Overview
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Brief Summary
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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.

Detailed Description
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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).

Official Title
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A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease

Conditions
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Recurrent Bacterial Infection, Autoimmune Disorder

Intervention / Treatment
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* Other: Natural History Observation


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Other: Natural History Observation|This is a natural history study.|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| General clinical condition | General clinical condition | Approximately every 3 months | 
| Proportion of patients with episodes or exacerbations of medical events | Proportion of patients with episodes or exacerbations of medical events | Approximately every 3 months | 
| Proportion of patients with infections As applicable, infections (particularly meningococcal and other encapsulated bacteria) | Proportion of patients with infections | Approximately every 3 months | 
| Proportion of patients with kidney disease progression | Proportion of patients with kidney disease progression | Approximately every 3 months |