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NADPH Oxidase Correction in Chronic Granulomatous Disease (CGD)

This study is testing a new procedure for men with Chronic Granulomatous Disease (CGD), a genetic condition where your body's cells can't effectively kill germs, leading to frequent infections. Researchers want to see if a treatment called gp91 Grans can help your cells kill germs. This involves taking some of your blood cells, adding messenger RNA (mRNA) to them to correct the gene mutation, and then giving these cells back to you as an intravenous (IV) infusion. The main goals are to find the highest safe dose of gp91 Grans and to make sure the procedure is practical and safe. You could be eligible if you are a male between 18 and 75 years old with CGD caused by a gp91phox gene mutation.

Study design
This is a Phase 1, open-label study involving 25 participants. It's a dose-escalation trial, meaning participants will receive increasing doses of gp91 Grans to find the safest and most effective amount.
What's involved
You will undergo screening tests including medical history, physical exam, blood and urine tests, and a strep throat swab. After receiving gp91 Grans, you will be hospitalized for at least 3 days and have regular blood collections.
Compensation
Not stated in the trial record.
Follow-up
You will return for a final study visit about 3 months after receiving the gp91 Grans infusion.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05189925

NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)

Recruiting
PHASE1Ages 18–75InterventionalTreatment
National Institute of Allergy and Infectious Diseases (NIAID)
~25 participants
Updated 2026-09-04 on ClinicalTrials.gov
What's tested:gp91 Grans

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Feasibility: Recruitment, implementation, and manufacturing of gp91-Grans for infusions.
Measured over 3 months
+2 more outcomes measured

Conditions

Chronic Granulomatous Disease
Infection

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NCT05189925

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • National Institutes of Health Clinical Center

    Bethesda, Marylandstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Suk S De Ravin, M.D. · PRINCIPAL_INVESTIGATOR · National Institute of Allergy and Infectious Diseases (NIAID)

Who to contact

Joanna L Peterson
[email protected]
(301) 346-9780
Email the study team

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Eligibility criteria

Inclusion

Males aged 18 to 75 years
CGD confirmed by DHR and deficiency subtype confirmed by protein analysis and/or genetic sequencing
Has a physician at home for follow-up care
Able to provide informed consent
For men who engage in activities that can result in pregnancy, agree to use contraception when engaging in sexual activities that can result in pregnancy. Contraception must be used from screening through 3 months after the CGD-Grans infusion. Acceptable methods of contraception include the following:
Hormonal contraception
Male or female condom

Exclusion

Clinically unstable due to moderate to severe acute systemic infections as defined by persistent resting tachypnea, tachycardia, or hypoxia of \>20% from baseline and hypotension.
Current or history of stage 4 chronic kidney disease or estimated glomerular filtration rate \[eGFR\] \<30 mL/min/1.73 m\^2 within 90 days of baseline.
Unstable diabetes mellitus with hemoglobin A1c \>7.0% and fasting serum glucose \>200 mg/dL at screening.
Current or history of heart failure stage D as defined by the American College of Cardiology Foundation/American Heart Association guidelines.
History of arrhythmias that are symptomatic and deemed clinically unsafe for participation by NIH CC Cardiology consultation.
Current or history of invasive cancers that require chemotherapy within 5 years of screening.
Active hepatitis B, C, or HIV infections at screening.
Unstable hypertension requiring addition of new anti-hypertensives within 2 weeks of screening.
Impaired renal function that is unstable, with serum creatinine \>3.0 mg/dL and rising.
Serum transaminases and bilirubin that are \>3 x the upper limit of normal.
Electrocardiogram abnormalities indicative of acute myocardial injury, or arrhythmias that presents anesthetic risks, at screening.
Anemia with hemoglobin \<8 g/dL (transfusions to correct anemia permitted).
Thrombocytopenia (platelets \<50 x10\^9 cells/L) (platelet transfusions to correct thrombocytopenia permitted).
Profound thrombocytopenia (platelet counts \<10,000/microliter) that is not reversible with platelet transfusions.
Abnormal prothrombin time/partial thromboplastin time (PT/PTT) values outside the ranges accepted at the NIH CC that are not corrected or that cannot be attributed to presence of Lupus anticoagulant (commonly found in CGD patients).
Inherited bleeding disorder that precludes line placement.
Severe oxygen-dependent pulmonary disease that increases risks of procedures that may require sedation.
History of or current evidence of alcohol or illicit drug abuse or dependence.
Participation in a clinical protocol that includes an intervention that, in the opinion of the investigator, may affect the results of the current study.

What this trial measures

  • Feasibility: Recruitment, implementation, and manufacturing of gp91-Grans for infusions.3 months

    Determine feasibility of gp91-Grans infusion.

  • MTD determination based on the rate of AEs. MTD is defined as the highest dose level that does not cause the same grade 3 or 4 AEs in 3 or more patients3 months

    Determine an MTD for administration.

  • Safety: Frequency of grade 3 or greater adverse events or serious adverse events related to the study agent3 months

    Determine safety of gp91-Grans infusion.