MRNA therapy gives immune cells a temporary boost against rare infection disease
NCT ID NCT05189925
First seen Jun 27, 2026 · Last updated Sep 04, 2026 · Updated 9 times
Summary
This early-stage trial tests a new approach for chronic granulomatous disease (CGD), a genetic condition that leaves people vulnerable to severe infections. Researchers take a participant's own white blood cells, use mRNA to correct the genetic defect, and infuse the cells back. The goal is to see if the procedure is safe and feasible, and whether it can temporarily restore the cells' ability to kill germs. The study involves 25 adult males and includes a hospital stay for cell collection and infusion.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- mRNA-corrected white blood cells (gp91 Grans)
- What this could lead to
- If successful, this could provide a temporary way to restore infection-fighting ability in people with CGD, reducing infection risk.
- What could go wrong
- This is a very early Phase 1 trial with only 25 participants. The effect is temporary, and there may be side effects from the infusion or the cell collection process.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
About 25 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Jul 2022
- Expected to finish
-
Jul 2027
An estimate. End dates often move.
- Lead sponsor
-
A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 75 years
- Sex
-
Male participants only
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* INCLUSION CRITERIA: Individuals must meet all of the following criteria to be eligible for study participation: * 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 CRITERIA: Individuals meeting any of the following criteria will be excluded from study participation: * 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. NOTE: For prospective subjects who, per PI assessment at screening, have abnormal liver function tests, and/or a significant history of liver disease, and/or liver-related complications of CGD, and who otherwise meet eligibility criteria \[i.e. those who do NOT meet any of the exclusion set forth herein\], a hepatology consult will be required at screening, and participation must be approved in writing by hepatology to the PI. * 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. Subjects will be selected in an equitable manner from the available pool of potentially eligible individuals, without regard to factors such as gender, race, ethnicity, socioeconomic status, etc, except for age and sex.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Chronic granulomatous disease are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The places running it
1 site. The list below names each one and where it is.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
-
National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a simple blood test predict sepsis two days early?
- Switching stomach drugs after transplant may tame a deadly immune attack
- Can a new antibody make stem cell transplants safer for CGD patients?
- Can telemedicine bring IV antibiotics home?
- Blood test may forecast recovery in elderly infection patients
- Aging in place: could mobile teams keep nursing home residents out of the hospital?