NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous … (NCT05189925) | Clinical Trial Compass
RecruitingPhase 1
NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
United States25 participantsStarted 2022-07-22
Plain-language summary
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.
Who can participate
Age range
18 Years – 75 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
* 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 Cardiol…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Feasibility: Recruitment, implementation, and manufacturing of gp91-Grans for infusions.
Timeframe: 3 months
2
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 patients
Timeframe: 3 months
3
Safety: Frequency of grade 3 or greater adverse events or serious adverse events related to the study agent
Timeframe: 3 months
Trial details
NCT IDNCT05189925
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)