TerminatedNot Applicable

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

Stopped: enrollment

United States3 participantsStarted 2021-08-31

Plain-language 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.

Who can participate

Age range6 Months

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

General clinical condition

Timeframe: Approximately every 3 months

Proportion of patients with episodes or exacerbations of medical events

Timeframe: Approximately every 3 months

Proportion of patients with infections As applicable, infections (particularly meningococcal and other encapsulated bacteria)

Timeframe: Approximately every 3 months

Proportion of patients with kidney disease progression

Timeframe: Approximately every 3 months

Trial details

NCT IDNCT05095623

SponsorCatalyst Biosciences

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2022-02-10

View on ClinicalTrials.gov More Recurrent Bacterial Infection, Autoimmune Disorder trials

Who can participate

Age range6 Months

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

General clinical condition

Timeframe: Approximately every 3 months

Proportion of patients with episodes or exacerbations of medical events

Timeframe: Approximately every 3 months

Proportion of patients with infections As applicable, infections (particularly meningococcal and other encapsulated bacteria)

Timeframe: Approximately every 3 months

Proportion of patients with kidney disease progression

Timeframe: Approximately every 3 months