A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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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).
Study Design
Outcome Measures
Primary Outcome Measures
- General clinical condition [Approximately every 3 months]
General clinical condition
- Proportion of patients with episodes or exacerbations of medical events [Approximately every 3 months]
Proportion of patients with episodes or exacerbations of medical events
- Proportion of patients with infections As applicable, infections (particularly meningococcal and other encapsulated bacteria) [Approximately every 3 months]
Proportion of patients with infections
- Proportion of patients with kidney disease progression [Approximately every 3 months]
Proportion of patients with kidney disease progression
Eligibility Criteria
Criteria
Inclusion Criteria:
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Male or female, age 6 months or older
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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.
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Confirmed CFI deficiency during the screening study, CFI-001, with a disease that is associated with an absence or deficiency in CFI
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Stated willingness of the participant to comply with all study procedures (including multiple blood draws) and availability for the duration of the study
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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:
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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
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Having a major concurrent non-CFI-related disease that prevents the assessment of the natural course of the CFI deficiency disease
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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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Nephrology Consultants, LLC | Huntsville | Alabama | United States | 35805 |
Sponsors and Collaborators
- Catalyst Biosciences
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CFI-002