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New treatment option for Chronic Granulomatous Disease
Official title NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
ClinicalTrials.gov ID: NCT05189925
What this study is testing
What is gp91 Grans?
gp91 Grans is an investigational medicine, given as an once-weekly infusion into a vein, being studied as a potential treatment for chronic granulomatous disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 75, men only
You may be able to join if
- 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
You likely can't join if
- 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%...
- 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.
See the full eligibility 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...
- Hormonal contraception
- Male or female condom
- 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 people who, per PI assessment at screening, have abnormal liver function tests, and/or a significant history of liver...
- 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...
- 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. people will be selected in an equitable manner from the available...
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 18 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.