A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Recurrent Bacterial Infection, Autoimmune Disorder
- Sponsor
- Catalyst Biosciences
- Enrollment
- 3
- Locations
- 1
- Primary Endpoint
- General clinical condition
- Status
- Terminated
- Last Updated
- 4 years ago
Overview
Brief Summary
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
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).
Investigators
Eligibility Criteria
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
Outcomes
Primary Outcomes
General clinical condition
Time Frame: Approximately every 3 months
General clinical condition
Proportion of patients with kidney disease progression
Time Frame: 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)
Time Frame: Approximately every 3 months
Proportion of patients with infections
Proportion of patients with episodes or exacerbations of medical events
Time Frame: Approximately every 3 months
Proportion of patients with episodes or exacerbations of medical events