Granulocyte transfusions in patients with chronic granulomatous disease and refractory infections: The NIH experience.
Beatriz E Marciano, Elisabeth S Allen, Cathy Conry-Cantilena...
https://pubmed.ncbi.nlm.nih.gov/28342916Actively Recruiting
Phase 1
Age: 18Years - 75Years
MALE
ID05189925
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
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating a new procedure to help cells in people with Chronic Granulomatous Disease (CGD) better fight infections. CGD is caused by a gene mutation that prevents cells from killing germs effectively, leading to frequent or serious infections. This phase 1, open-label trial aims to assess the safety and feasibility of giving patients with a specific CGD gene mutation a treatment involving messenger RNA (mRNA) to correct their blood cells temporarily. The treatment involves collecting granulocyte-enriched white blood cells from each participant through a procedure called apheresis. These cells are then treated with mRNA to correct the gene defect and infused back into the participant through an intravenous (IV) line. The trial uses a dose-escalation design with three doses: 1 million, 10 million, and up to 500 million cells per kilogram of body weight, given to different groups to find the safest and most effective dose. Participants receive one infusion and are monitored in the hospital for at least three days afterward. Participants will undergo medical exams, blood and urine tests, and throat swabs during screening and throughout the study. After hospital discharge, they will keep a symptom diary and have weekly contact for one month, then monthly follow-up visits including a final visit three months after infusion. Researchers will measure safety by tracking serious side effects, determine the maximum tolerated dose, and assess how well the corrected cells function and persist in the blood. The total study duration for each participant is about three months after treatment.
CONDITIONS
Brief Title
NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
Who Can Participate
Age: 18Years - 75Years
MALE
Eligibility Criteria
You may qualify if you...
- Male aged 18 to 75 years
- Diagnosis of CGD confirmed by DHR test and protein or genetic analysis
- Has a physician at home for follow-up care
- Able to provide informed consent
- Agree to use contraception if engaging in activities that can result in pregnancy from screening through 3 months after infusion, using hormonal contraception or condoms
You will not qualify if you...
- Moderate to severe acute systemic infections causing significant breathing, heart rate, oxygen, or blood pressure problems
- Current or history of stage 4 chronic kidney disease or eGFR below 30 mL/min/1.73 m2 within 90 days
- 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 deemed unsafe
- Invasive cancers requiring chemotherapy within 5 years
- Active hepatitis B, C, or HIV infections
- Unstable hypertension needing new medication within 2 weeks
- Unstable impaired kidney function with rising serum creatinine over 3.0 mg/dL
- Liver enzyme levels over 3 times normal
- Abnormal ECG showing acute injury or risky arrhythmias
- Anemia with hemoglobin below 8 g/dL (unless corrected)
- Thrombocytopenia with platelets below 50 x10^9/L (unless corrected)
- Severe thrombocytopenia under 10,000/microliter not corrected by transfusion
- Abnormal clotting times not corrected or linked to Lupus anticoagulant
- Inherited bleeding disorder preventing line placement
- Severe oxygen-dependent lung disease raising sedation risks
- History or current alcohol or drug abuse
- Participating in another intervention trial that may affect results
- Abnormal liver function or liver disease requiring hepatology approval
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Hospitalization for at least 3 days after infusion
Participants undergo granulocyte-enriched apheresis to provide cells for mRNA correction and then receive 1 intravenous infusion of CGD-Grans at an escalating dose. Participants are hospitalized for at least 3 days following the infusion for monitoring.
1 hospitalization period and regular blood collection during stay
Follow-up
Duration - Approximately 3 months
Participants return for a final study visit about 3 months after the infusion for safety and efficacy evaluations including blood collection.
1 final follow-up visit approximately 3 months after treatment
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
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Research Team
J
Joanna L Peterson
S
Suk S De Ravin, M.D.
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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Published Research Related To This Trial
Alloimmunization after granulocyte transfusions.
D F Stroncek, K Leonard, G Eiber...
https://pubmed.ncbi.nlm.nih.gov/8937413The relationship between alloimmunization and posttransfusion granulocyte survival: experience in a chronic granulomatous disease cohort.
K F Heim, T A Fleisher, D F Stroncek...
https://pubmed.ncbi.nlm.nih.gov/21175646