A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease

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.

Study Overview

• Status: Terminated
• Conditions: Recurrent Bacterial Infection, Autoimmune Disorder
• Intervention / Treatment: Other: Natural History Observation

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 Type: Observational
• Enrollment (Actual): 3

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Huntsville, Alabama, United States, 35805
      • Nephrology Consultants, LLC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

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).

Description

Inclusion Criteria:

1. Male or female, age 6 months or older
2. 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.
3. Confirmed CFI deficiency during the screening study, CFI-001, with a disease that is associated with an absence or deficiency in CFI
4. Stated willingness of the participant to comply with all study procedures (including multiple blood draws) and availability for the duration of the study
5. 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:

1. 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
2. Having a major concurrent non-CFI-related disease that prevents the assessment of the natural course of the CFI deficiency disease
3. 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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
General clinical condition	General clinical condition	Approximately every 3 months
Proportion of patients with episodes or exacerbations of medical events	Proportion of patients with episodes or exacerbations of medical events	Approximately every 3 months
Proportion of patients with infections As applicable, infections (particularly meningococcal and other encapsulated bacteria)	Proportion of patients with infections	Approximately every 3 months
Proportion of patients with kidney disease progression	Proportion of patients with kidney disease progression	Approximately every 3 months

Collaborators and Investigators

• Sponsor: Catalyst Biosciences

Study record dates

Study Major Dates

• Study Start (Actual): August 31, 2021
• Primary Completion (Actual): February 10, 2022
• Study Completion (Actual): February 10, 2022

Study Registration Dates

• First Submitted: September 29, 2021
• First Submitted That Met QC Criteria: October 15, 2021
• First Posted (Actual): October 27, 2021

Study Record Updates

• Last Update Posted (Actual): April 8, 2022
• Last Update Submitted That Met QC Criteria: March 31, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Infections; Immune System Diseases; Bacterial Infections and Mycoses; Autoimmune Diseases; Bacterial Infections
• Other Study ID Numbers: CFI-002

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No