A Natural History Study to Assess the Clinical Outcomes o... | NCT05095623

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

NCT05095623

Sponsor

Catalyst Biosciences

Status

Terminated

Last Update Posted

Apr 8, 2022Actual

Enrollment

3Actual

Phase

Not provided

Conditions

Recurrent Bacterial Infection, Autoimmune Disorder

Interventions

Natural History Observation

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2022

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

enrollment

Expanded Access Info

Start Date

Aug 31, 2021Actual

Primary Completion Date

Feb 10, 2022Actual

Completion Date

Feb 10, 2022Actual

First Submitted Date

Sep 29, 2021

First Submission Date that Met QC Criteria

Oct 15, 2021

First Posted Date

Oct 27, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 31, 2022

Last Update Posted Date

Apr 8, 2022Actual

Description Module

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.

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).

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

3Actual

Name	Type	Description	Arm Group Labels	Other Names
Natural History Observation	Other	This is a natural history study.

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Months

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

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).

Sampling Method

Non-Probability Sample

ID	Term
D001424	Bacterial Infections
D001327	Autoimmune Diseases

ID	Term
D001423	Bacterial Infections and Mycoses
D007239	Infections
D007154	Immune System Diseases