Recurrent Bacterial Infection, Autoimmune DisorderNCT05095623
A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease
An observational study of Recurrent Bacterial Infection, Autoimmune Disorder, run by Catalyst Biosciences at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Recurrent Bacterial Infection, Autoimmune Disorder
- What is being tested?
- Natural History Observation
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Aug 2021to 10 Feb 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older 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).
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Nephrology Consultants, LLC
Huntsville, Alabama, 35805
About this study
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.
Read the 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).
- Being tested
- Natural History Observation
- Medical subject headings
- Bacterial Infections, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Natural History ObservationOther
This is a natural history study.
What is being measured
The primary outcome is the main question the study is designed to answer.
General clinical condition
General clinical condition
Measured: Approximately every 3 months
Proportion of patients with episodes or exacerbations of medical events
Proportion of patients with episodes or exacerbations of medical events
Measured: Approximately every 3 months
Proportion of patients with infections As applicable, infections (particularly meningococcal and other encapsulated bacteria)
Proportion of patients with infections
Measured: Approximately every 3 months
Proportion of patients with kidney disease progression
Proportion of patients with kidney disease progression
Measured: Approximately every 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2021
- Study start31 Aug 2021
- Primary completion10 Feb 2022
- Study completion10 Feb 2022
Why it stopped: enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recurrent Bacterial Infection, Autoimmune DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Catalyst Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05095623
- Sponsor's protocol ID
- CFI-002
- Status last verified
- Mar 2022
- First submitted
- 29 Sep 2021
- Last updated
- 8 Apr 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease
- ClinicalTrials.gov ID
- NCT05095623
- Acronym / sponsor ID
- CFI-002
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: enrollment
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Catalyst BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 31 Aug 2021 (actual)
- Primary completion
- 10 Feb 2022 (actual)
- Study completion
- 10 Feb 2022 (actual)
- First posted
- 27 Oct 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older 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).
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Natural History ObservationThis is a natural history study. | Other | – |
Primary outcome
General clinical condition
Time frame: Approximately every 3 months
General clinical condition
Proportion of patients with episodes or exacerbations of medical events
Time frame: Approximately every 3 months
Proportion of patients with episodes or exacerbations of medical events
Proportion of patients with infections As applicable, infections (particularly meningococcal and other encapsulated bacteria)
Time frame: Approximately every 3 months
Proportion of patients with infections
Proportion of patients with kidney disease progression
Time frame: Approximately every 3 months
Proportion of patients with kidney disease progression
Results and safety
The study was stopped early (reason given: enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Recurrent Bacterial Infection, Autoimmune Disorder, run by Catalyst Biosciences at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: enrollment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
General clinical condition (time frame: Approximately every 3 months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2022.