Previous Diagnosis With a Complement-mediated Disease and/or With Clinical Manifestations Reasonably Associated With Complement Factor I Deficiency
Conditions
Brief summary
This is screening study to identify patients with Complement Factor I deficiencies. The primary objective is to identify participants with CFI deficiencies and assess the prevalence in the screened population.
Interventions
None listed
Sponsors
Catalyst Biosciences
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Months to No maximum
Healthy volunteers
No
Inclusion criteria
An individual must meet all the following criteria to be eligible to participate in this study: 1. A male or female ≥6 months of age who has been diagnosed with a complement-mediated disease in which a reasonable etiological association with CFI deficiency has been reported 2. Affirmation of participant's informed consent or 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 be willing to give written informed assent if the minor is within the age groups 7 to 11 years old and 12 to 17 years old.
Exclusion criteria
1\) Diagnosis of age-related macular degeneration that is complement-related with no concurrent systemic complement involvement
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Complement Factor I Deficiency Identification | At Screening | Identification of participants with a CFI deficiency and percentage of those with a CFI deficiency versus total number of screened participants |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Demographics, Disease Characteristics, Medication Use | At Screening | Demographics, disease characteristics (including disease history), and disease-related medication use. Complement levels and mutation data will also be collected from medical records, if previously assessed and available. |
Countries
United States
Outcome results
None listed