Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease

NCT06605378 | Recruiting

• 1 other identifier: EN-374-100
• Study Type: observational
• Target: 60
• Locations: 2 countries
• Sites: 52

Brief Summary

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

Conditions

Chronic Granulomatous Disease (CGD)

Keywords

CGD; X-CGD; Chronic Granulomatous Disease; Granulomatous Disease, Chronic; Autosomal Recessive Chronic Granulomatous Disease

Outcome Measures

Primary Outcomes (2)

• Total adenoviral-specific antibody (TAb) titer

  Level of total adenoviral-specific antibody in blood

  1 Day

• Adenoviral-specific neutralizing antibody (NAb) titer

  Level of adenoviral-specific neutralizing antibodies in blood

  1 Day

Secondary Outcomes (1)

• Circulating inflammatory cytokine levels

  1 Day

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

People with CGD

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Ensoma: lead

Study Sites (52)

Home-based telemedicine

Montgomery, Alabama, 36104, United States

RECRUITING

Home-based telemedicine

Phoenix, Arizona, 85001, United States

RECRUITING

Home-based telemedicine

Little Rock, Arkansas, 72201, United States

RECRUITING

Home-based telemedicine

Sacramento, California, 95814, United States

RECRUITING

University of California, San Francisco

San Francisco, California, 94158, United States

RECRUITING

Home-based telemedicine

Denver, Colorado, 80202, United States

RECRUITING

Home-based telemedicine

Hartford, Connecticut, 06103, United States

RECRUITING

Home-based telemedicine

Dover, Delaware, 19901, United States

RECRUITING

Home-based telemedicine

Washington D.C., District of Columbia, 20001, United States

RECRUITING

Home-based telemedicine

Tallahassee, Florida, 32301, United States

RECRUITING

Home-based telemedicine

Atlanta, Georgia, 30303, United States

RECRUITING

Home-based telemedicine

Boise, Idaho, 83702, United States

RECRUITING

Home-based telemedicine

Springfield, Illinois, 62701, United States

RECRUITING

Home-based telemedicine

Indianapolis, Indiana, 46225, United States

RECRUITING

Home-based telemedicine

Des Moines, Iowa, 50309, United States

RECRUITING

Home-based telemedicine

Topeka, Kansas, 66603, United States

RECRUITING

Home-based telemedicine

Frankfort, Kentucky, 40601, United States

RECRUITING

Home-based telemedicine

Baton Rouge, Louisiana, 70802, United States

RECRUITING

Home-based telemedicine

Augusta, Maine, 04330, United States

RECRUITING

Home-based telemedicine

Annapolis, Maryland, 21401, United States

RECRUITING

Home-based telemedicine

Boston, Massachusetts, 02210, United States

RECRUITING

Home-based telemedicine

Lansing, Michigan, 48933, United States

RECRUITING

Home-based telemedicine

Saint Paul, Minnesota, 55102, United States

RECRUITING

Home-based telemedicine

Jackson, Mississippi, 39205, United States

RECRUITING

Home-based telemedicine

Jefferson City, Missouri, 65101, United States

RECRUITING

Home-based telemedicine

Helena, Montana, 59623, United States

RECRUITING

Home-based telemedicine

Lincoln, Nebraska, 68502, United States

RECRUITING

Home-based telemedicine

Carson City, Nevada, 89701, United States

RECRUITING

Home-based telemedicine

Concord, New Hampshire, 03301, United States

RECRUITING

Home-based telemedicine

Trenton, New Jersey, 08608, United States

RECRUITING

Home-based telemedicine

Santa Fe, New Mexico, 87501, United States

RECRUITING

Home-based telemedicine

Albany, New York, 12207, United States

RECRUITING

Science 37, Inc

Morrisville, North Carolina, 27560, United States

RECRUITING

Home-based telemedicine

Bismarck, North Dakota, 58501, United States

RECRUITING

Home-based telemedicine

Columbus, Ohio, 43215, United States

RECRUITING

Home-based telemedicine

Oklahoma City, Oklahoma, 73102, United States

RECRUITING

Home-based telemedicine

Salem, Oregon, 97301, United States

RECRUITING

Home-based telemedicine

Harrisburg, Pennsylvania, 17101, United States

RECRUITING

Home-based telemedicine

Providence, Rhode Island, 02903, United States

RECRUITING

Home-based telemedicine

Columbia, South Carolina, 29217, United States

RECRUITING

Home-based telemedicine

Pierre, South Dakota, 57501, United States

RECRUITING

Home-based telemedicine

Nashville, Tennessee, 37219, United States

RECRUITING

Home-based telemedicine

Austin, Texas, 78701, United States

RECRUITING

Home-based telemedicine

Salt Lake City, Utah, 84111, United States

RECRUITING

Home-based telemedicine

Montpelier, Vermont, 05602, United States

RECRUITING

Home-based telemedicine

Richmond, Virginia, 23219, United States

RECRUITING

Home-based telemedicine

Olympia, Washington, 98507, United States

RECRUITING

Home-based telemedicine

Charleston, West Virginia, 25301, United States

RECRUITING

Home-based telemedicine

Madison, Wisconsin, 53703, United States

RECRUITING

Home-based telemedicine

Cheyenne, Wyoming, 82001, United States

RECRUITING

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

COMPLETED

Royal Free London NHS Foundation Trust

London, United Kingdom

COMPLETED

Biospecimen

Retention: SAMPLES WITH DNA

Blood

MeSH Terms

Conditions

Granulomatous Disease, Chronic

Condition Hierarchy (Ancestors)

Phagocyte Bactericidal Dysfunction; Leukocyte Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Andrew Dietz, MD, MSCR

CONTACT

617-766-3917; ddietz@ensoma.com

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2024
• First Posted: September 20, 2024
• Study Start: November 18, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: March 3, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2); US (50)