Actively Recruiting
NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
Led by National Institute of Allergy and Infectious Diseases (NIAID) · Updated on 2026-04-24
25
Participants Needed
1
Research Sites
205 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: CGD is caused by a gene mutation. For people with CGD, their cells cannot kill germs well, so they can get frequent or life-threatening infections. Researchers want to see if a new procedure can help a person s cells kill germs for a short time. It uses messenger RNA (mRNA) to deliver correct instructions for the gene mutation to the cells. Objective: To test a procedure in which mRNA is added to a person s blood cells. Eligibility: Males aged 18-75 with CGD with a mutation in the gene that makes the protein gp91phox. Design: Participants will be screened with: Medical history Physical exam Blood and urine tests Swab to test for strep throat Some screening tests will be repeated during the study. Participants will be admitted to the NIH Clinical Center hospital for at least 7 days. They will have apheresis. For this, a medicine is injected under their skin to prepare their white blood cells for collection. An IV line is placed into an arm vein. Blood goes through the IV line into a machine that divides whole blood into red blood cells, plasma, and white blood cells. The white blood cells are removed, and the rest of the blood is returned to the participant through an IV line in their other arm. The next day, they will get their mRNA-corrected cells via IV. They will be monitored for 3 more days. After discharge, participants will keep a symptom diary. They will be contacted weekly for one month, and then once a month. They will have a follow-up visit 3 months after the infusion.
CONDITIONS
Official Title
NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male aged 18 to 75 years
- Confirmed diagnosis of CGD with deficiency subtype verified by protein analysis or genetic sequencing
- Has a physician available for follow-up care at home
- Able to give informed consent
- Agree to use contraception from screening through 3 months after infusion if sexually active and capable of causing pregnancy, using hormonal contraception or condoms
You will not qualify if you...
- Unstable due to moderate to severe acute systemic infections with symptoms like persistent rapid breathing, fast heartbeat, hypoxia over 20% from baseline, or low blood pressure
- Current or history of stage 4 chronic kidney disease or eGFR below 30 mL/min/1.73 m² within 90 days of baseline
- Unstable diabetes with hemoglobin A1c over 7.0% and fasting glucose over 200 mg/dL
- Current or history of stage D heart failure
- Symptomatic arrhythmias considered unsafe by cardiology
- Current or history of invasive cancers requiring chemotherapy within 5 years
- Active hepatitis B, C, or HIV infections at screening
- Unstable hypertension needing new medications within 2 weeks of screening
- Unstable impaired renal function with serum creatinine over 3.0 mg/dL
- Elevated liver enzymes or bilirubin over 3 times normal limits
- Abnormal ECG findings indicating acute myocardial injury or risky arrhythmias
- Anemia with hemoglobin less than 8 g/dL (transfusions allowed)
- Thrombocytopenia with platelets below 50 x10⁹ cells/L (transfusions allowed)
- Severe thrombocytopenia below 10,000/microliter not reversible by transfusions
- Abnormal clotting times not corrected or attributable to lupus anticoagulant
- Inherited bleeding disorders preventing line placement
- Severe oxygen-dependent lung disease increasing sedation risk
- History or current alcohol or drug abuse
- Participation in other clinical protocols that might interfere with this study's results
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
J
Joanna L Peterson
CONTACT
S
Suk S De Ravin, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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