Screening Study to Identify Patients With Complement Factor I Deficiencies

Terminated

• Conditions: Previous Diagnosis With a Complement-mediated Disease and/or With Clinical Manifestations Reasonably Associated With Complement Factor I Deficiency

• Registration Number: NCT05072912
• Lead Sponsor: Catalyst Biosciences

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-07-21
• Study First Submitted: 2021-09-29
• Study First Submitted QC: 2021-09-29
• Study First Posted: 2021-10-11
• Primary Completion: 2021-11-23
• Study Completion: 2021-11-23
• Status Verified: 2022-03
• Last Update Submitted: 2022-03-31
• Last Update Posted: 2022-04-08

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 113

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Nephrology Consultants, LLC; 🇺🇸 Huntsville, Alabama, United States