Screening Study to Identify Patients With Complement Factor I Deficiencies

NCT05072912 | Terminated

• 1 other identifier: CFI-001
• Study Type: observational
• Target: 113
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Complement Factor I Deficiency Identification

  Identification of participants with a CFI deficiency and percentage of those with a CFI deficiency versus total number of screened participants

  At Screening

Secondary Outcomes (1)

• Demographics, Disease Characteristics, Medication Use

  At Screening

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients previously diagnosed with a complement-mediated disease and/or with clinical manifestations in which a reasonable etiological association with Complement Factor I (CFI) deficiency has been reported. Examples of these diseases and conditions are (but not limited to): atypical hemolytic uremic syndrome, Complement 3 Glomerulopathy, membranoproliferative glomerulonephritis, systemic lupus erythematosus, leukocytoclastic vasculitis, recurrent bacterial infections, unexplained inflammation or edema, and some autoimmune disorders.

You may qualify if:

• An individual must meet all the following criteria to be eligible to participate in this study:
• 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
• 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.

You may not qualify if:

• \) Diagnosis of age-related macular degeneration that is complement-related with no concurrent systemic complement involvement

Sponsors & Collaborators

• Catalyst Biosciences: lead

Study Sites (1)

Nephrology Consultants, LLC

Huntsville, Alabama, 35805, United States

Location

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 29, 2021
• First Posted: October 11, 2021
• Study Start: July 21, 2021
• Primary Completion: November 23, 2021
• Study Completion: November 23, 2021
• Last Updated: April 8, 2022

Record last verified: 2022-03

Locations

US (1)