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

NCT05095623 | Terminated

• 1 other identifier: CFI-002
• Study Type: observational
• Target: 3
• Locations: 1 country
• Sites: 1

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.

Conditions

Recurrent Bacterial Infection, Autoimmune Disorder

Outcome Measures

Primary Outcomes (4)

• 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

Interventions

Natural History Observation OTHER

This is a natural history study.

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

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).

You may qualify if:

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

You may not qualify if:

• 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
• Having a major concurrent non-CFI-related disease that prevents the assessment of the natural course of the CFI deficiency disease
• 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

Sponsors & Collaborators

• Catalyst Biosciences: lead

Study Sites (1)

Nephrology Consultants, LLC

Huntsville, Alabama, 35805, United States

Location

MeSH Terms

Conditions

Bacterial Infections; Autoimmune Diseases

Condition Hierarchy (Ancestors)

Bacterial Infections and Mycoses; Infections; Immune System Diseases

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 27, 2021
• Study Start: August 31, 2021
• Primary Completion: February 10, 2022
• Study Completion: February 10, 2022
• Last Updated: April 8, 2022

Record last verified: 2022-03

Locations

US (1)