Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease

Recruiting

• Conditions: Chronic Granulomatous Disease (CGD)

• Registration Number: NCT06605378
• Lead Sponsor: Ensoma

Brief Summary

This research study is designed to assess the prevalence of specific antibodies and inflammatory cytokines in adult and pediatric participants with CGD.

Detailed Description

In this observational study, blood samples will be collected from adult and pediatric patients with CGD. The level of circulating adenovirus antibodies and inflammatory cytokines will be measured to characterize the prevalence of these markers in this population.

Study Timeline

• Study First Submitted: 2024-09-10
• Study First Submitted QC: 2024-09-19
• Study First Posted: 2024-09-20
• Study Start: 2024-11-18
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-07
• Last Update Posted: 2025-02-11
• Primary Completion: 2025-03
• Study Completion: 2025-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

• The participant must have been diagnosed with CGD based on the referring physician's confirmation that NADPH oxidase activity is ≤5% (i.e., the percentage of dihydrorhodamine-positive [DHR+] cells is ≤5% by flow cytometry) OR based on confirmed pathogenic mutation in a CGD associated gene (CYBB, CYBA, NCF1, NCF2, NCF4, or CYBC1).
• The participant or the participant's legally authorized guardian or representative (if applicable) must be capable of giving signed informed consent.
• The participant (with assistance from the participant's legally authorized guardian/representative or primary caregiver, if applicable) must be capable of complying with the requirements and restrictions listed in the protocol and informed consent form (ICF).

Exclusion Criteria

• The participant has undergone an allogeneic bone marrow transplant or investigational gene therapy.
• The participant is unable to comply with the sample collection procedure based on investigator judgment.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Total adenoviral-specific antibody (TAb) titer	1 Day	Level of total adenoviral-specific antibody in blood
Adenoviral-specific neutralizing antibody (NAb) titer	1 Day	Level of adenoviral-specific neutralizing antibodies in blood

Secondary Outcome Measures

Name	Time	Method
Circulating inflammatory cytokine levels	1 Day	Level of inflammatory cytokines in blood

Trial Locations

Locations (5)

Science 37, Inc; 🇺🇸 Morrisville, North Carolina, United States

University of California, San Francisco; 🇺🇸 San Francisco, California, United States

Home-based telemedicine; 🇺🇸 Cheyenne, Wyoming, United States

Royal Free London NHS Foundation Trust; 🇬🇧 London, United Kingdom

Great Ormond Street Hospital for Children; 🇬🇧 London, United Kingdom