Chronic Granulomatous DiseasePhase 1NCT05189925
NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
A Phase 1 study testing gp91 Grans in people with Chronic Granulomatous Disease and Infection, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years, men only
- Conditions the study is for
- Chronic Granulomatous Disease, Infection
- What is being tested?
- gp91 Grans
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Jul 2022to 1 Jul 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Show all 22Show fewer
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR), 800-411-1222 ext. TTY dial 711, ccopr@nih.gov
About this study
Official title: NADPH Oxidase Correction in mRNA Transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
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.
Read more
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....
Read the detailed description
Study Design:
This is a phase 1, open-label, dose-escalation trial to assess the safety and feasibility of administering CGD-Grans to adult patients with X-linked CGD and to identify the maximum tolerated dose (MTD). Subjects will undergo granulocyte-enriched apheresis to provide cell product for mRNA-correction and then receive 1 administration of study agent. The first subjects enrolled will receive the study agent at the lowest dose, and when a level has been determined to be safe, the dose level will be increased to a second and then third dose level for subsequent subjects.Subjects will be hospitalized for at least 3 days after study agent administration and will return for a final study visit about 3 months after administration. Blood will be collected regularly for safety and research evaluations. The study hypothesis is that it is safe and feasible to administer mRNA-transfected autologous granulocyte-enriched apheresis product to restore protein expression and phagocyte nicotinamide adenine dinucleotide phosphate (NADPH) oxidase function in patients with CGD.
Study Agent Description:
The study agent is one administration of autologous CGD mRNA transfected granulocyte-enriched cells, referred to as CGD-Grans. The study agent is derived from apheresis product enriched for granulocytes, which are then transfected with CGD mRNA and administered as an intravenous (IV) infusion. CGD-Grans will be evaluated using the 3+3 modified Fibonacci model at the following 3 escalating dosages:
Dose K: 1 x 10^6 granulocyte-enriched cells/kg body mass
Dose K+1: 1 x 10^7 granulocyte-enriched cells/kg body mass
Dose K+2: 1-5 x 10^8 granulocyte-enriched cells/kg body mass
Each dose will be evaluated in at least 3 subjects. Dose escalation will be managed by a predetermined algorithm depending on the occurrence of drug-related toxicity (DRT). There will be at least 1 week between study agent administration to each subject.
Primary Objectives:
- Determine the safety and feasibility of CGD-Grans infusion.
- Determine an MTD for administration.
Secondary Objectives:
- Assess efficacy of CGD-Grans at restoring NADPH oxidase.
- Determine the kinetics of CGD-Grans.
Exploratory Objectives:
- Assess for inflammatory responses to CGD-Grans infusion.
- Assess for immune responses to protein expressed by the transfected mRNA.
- Evaluate in vitro bactericidal activity of CGD-Grans.
Primary Endpoints:
- Determine safety and feasibility by: Safety: Frequency of grade 3 or greater adverse events (AEs) or serious adverse events (SAEs) related to the study agent. Feasibility: Recruitment, implementation, and manufacturing of CGD-Grans for infusions.
- 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.
Secondary Endpoints:
- Determine percent of circulating dihydrorhodamine (DHR) positive granulocytes following study agent infusion.
- Serial measurement of circulating DHR+ granulocytes from peripheral blood until day 3 following study agent infusion or disappearance of DHR+ granulocytes.
Exploratory Endpoint:
- Assess for increased expression of inflammation-related genes after study agent infusion.
- Evaluate for development of antibodies against mRNA-expressed CGDphox.
- Perform in vitro bactericidal activity of CGD-Grans.
- Conditions
- Chronic Granulomatous Disease, Infection
- Being tested
- gp91 Grans
- Keywords
- Primary Immune Deficiency, systemic infection, autologous transfusion, Apheresis
- Medical subject headings
- Granulomatous Disease, Chronic, Infections, Primary Immunodeficiency Diseases, Toxemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
gp91 GransBiological
Adults with gp91phox-deficient CGD without systemic infection will participate in a dose escalation trial to identify the MTD (the most effective yet safe dose) of gp91 Grans IV infusion.
| Study group | Role | What they receive |
|---|---|---|
| IV infusion of gp91-Grans at dose K: 1e6 cells/kg | Experimental | Adult CGD patients without systemic infection will participate in a dose-escalation trial to identify the most effective yet safe dose of study agent. Subjects enrolled will receive 1 administration of study agent at dose K, and safety of dose will be determined. Receives: Biological: gp91 Grans |
| IV infusion of gp91-Grans at dose K+1:1e7 cells/kg | Experimental | Adult CGD patients without systemic infection will participate in a dose-escalation trial to identify the most effective yet safe dose of study agent. Subjects enrolled will receive 1 administration of study agent at dose K+1, and safety of dose will be determined. Receives: Biological: gp91 Grans |
| IV infusion of gp91-Grans at dose K+2: 1-5e8 cells/kg | Experimental | Adult CGD patients without systemic infection will participate in a dose-escalation trial to identify the most effective yet safe dose of study agent. Subjects enrolled will receive 1 administration of study agent at dose K+2, and safety of dose will be determined. Receives: Biological: gp91 Grans |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility: Recruitment, implementation, and manufacturing of gp91-Grans for infusions.
Determine feasibility of gp91-Grans infusion.
Measured: 3 months
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
Determine an MTD for administration.
Measured: 3 months
Safety: Frequency of grade 3 or greater adverse events or serious adverse events related to the study agent
Determine safety of gp91-Grans infusion.
Measured: 3 months
Secondary outcomes (2)
Determine percent of circulating dihydrorhodamine (DHR) positive granulocytes following study agent infusion.
Assess efficacy of gp91-Grans at restoring NADPH oxidase
Measured: 3 months
Serial measurement of circulating DHR+ granulocytes from peripheral blood until day 3 following study agent infusion or disappearance of DHR+ granulocytes.
Determine the kinetics of gp91-Grans.
Measured: 3 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2022
- Study start22 Jul 2022
- Primary completion1 Jul 2027 (estimated)
- Study completion1 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Chronic Granulomatous DiseaseStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Suk S De Ravin, M.D., Principal investigator, National Institute of Allergy and Infectious Diseases (NIAID)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05189925
- Sponsor's protocol ID
- 220001
- Other IDs
- 22-I-0001
- Status last verified
- 29 Sep 2026
- First submitted
- 12 Jan 2022
- Last updated
- 29 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Marciano BE, Allen ES, Conry-Cantilena C, Kristosturyan E, Klein HG, Fleisher TA, Holland SM, Malech HL, Rosenzweig SD. Granulocyte transfusions in patients with chronic granulomatous disease and refractory infections: The NIH experience. J Allergy Clin Immunol. 2017 Aug;140(2):622-625. doi: 10.1016/j.jaci.2017.02.026. Epub 2017 Mar 22.
PubMed 28342916Stroncek DF, Leonard K, Eiber G, Malech HL, Gallin JI, Leitman SF. Alloimmunization after granulocyte transfusions. Transfusion. 1996 Nov-Dec;36(11-12):1009-15. doi: 10.1046/j.1537-2995.1996.36111297091747.x.
PubMed 8937413Heim KF, Fleisher TA, Stroncek DF, Holland SM, Gallin JI, Malech HL, Leitman SF. The relationship between alloimmunization and posttransfusion granulocyte survival: experience in a chronic granulomatous disease cohort. Transfusion. 2011 Jun;51(6):1154-62. doi: 10.1111/j.1537-2995.2010.02993.x. Epub 2010 Dec 22.
PubMed 21175646
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- NADPH Oxidase Correction in mRNA Transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
- ClinicalTrials.gov ID
- NCT05189925
- Acronym / sponsor ID
- 220001
- Conditions
- Chronic Granulomatous Disease, Infection
- Keywords
- Primary Immune Deficiency, systemic infection, autologous transfusion, Apheresis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Study start
- 22 Jul 2022 (actual)
- Primary completion
- 1 Jul 2027 (estimated)
- Study completion
- 1 Jul 2027 (estimated)
- First posted
- 13 Jan 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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....
Detailed description
Study Design:
This is a phase 1, open-label, dose-escalation trial to assess the safety and feasibility of administering CGD-Grans to adult patients with X-linked CGD and to identify the maximum tolerated dose (MTD). Subjects will undergo granulocyte-enriched apheresis to provide cell product for mRNA-correction and then receive 1 administration of study agent. The first subjects enrolled will receive the study agent at the lowest dose, and when a level has been determined to be safe, the dose level will be increased to a second and then third dose level for subsequent subjects.Subjects will be hospitalized for at least 3 days after study agent administration and will return for a final study visit about 3 months after administration. Blood will be collected regularly for safety and research evaluations. The study hypothesis is that it is safe and feasible to administer mRNA-transfected autologous granulocyte-enriched apheresis product to restore protein expression and phagocyte nicotinamide adenine dinucleotide phosphate (NADPH) oxidase function in patients with CGD.
Study Agent Description:
The study agent is one administration of autologous CGD mRNA transfected granulocyte-enriched cells, referred to as CGD-Grans. The study agent is derived from apheresis product enriched for granulocytes, which are then transfected with CGD mRNA and administered as an intravenous (IV) infusion. CGD-Grans will be evaluated using the 3+3 modified Fibonacci model at the following 3 escalating dosages:
Dose K: 1 x 10^6 granulocyte-enriched cells/kg body mass
Dose K+1: 1 x 10^7 granulocyte-enriched cells/kg body mass
Dose K+2: 1-5 x 10^8 granulocyte-enriched cells/kg body mass
Each dose will be evaluated in at least 3 subjects. Dose escalation will be managed by a predetermined algorithm depending on the occurrence of drug-related toxicity (DRT). There will be at least 1 week between study agent administration to each subject.
Primary Objectives:
- Determine the safety and feasibility of CGD-Grans infusion.
- Determine an MTD for administration.
Secondary Objectives:
- Assess efficacy of CGD-Grans at restoring NADPH oxidase.
- Determine the kinetics of CGD-Grans.
Exploratory Objectives:
- Assess for inflammatory responses to CGD-Grans infusion.
- Assess for immune responses to protein expressed by the transfected mRNA.
- Evaluate in vitro bactericidal activity of CGD-Grans.
Primary Endpoints:
- Determine safety and feasibility by: Safety: Frequency of grade 3 or greater adverse events (AEs) or serious adverse events (SAEs) related to the study agent. Feasibility: Recruitment, implementation, and manufacturing of CGD-Grans for infusions.
- 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.
Secondary Endpoints:
- Determine percent of circulating dihydrorhodamine (DHR) positive granulocytes following study agent infusion.
- Serial measurement of circulating DHR+ granulocytes from peripheral blood until day 3 following study agent infusion or disappearance of DHR+ granulocytes.
Exploratory Endpoint:
- Assess for increased expression of inflammation-related genes after study agent infusion.
- Evaluate for development of antibodies against mRNA-expressed CGDphox.
- Perform in vitro bactericidal activity of CGD-Grans.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| IV infusion of gp91-Grans at dose K: 1e6 cells/kg | Experimental | Biological: gp91 GransAdult CGD patients without systemic infection will participate in a dose-escalation trial to identify the most effective yet safe dose of study agent. Subjects enrolled will receive 1 administration of study agent at dose K, and safety of dose will be determined. |
| IV infusion of gp91-Grans at dose K+1:1e7 cells/kg | Experimental | Biological: gp91 GransAdult CGD patients without systemic infection will participate in a dose-escalation trial to identify the most effective yet safe dose of study agent. Subjects enrolled will receive 1 administration of study agent at dose K+1, and safety of dose will be determined. |
| IV infusion of gp91-Grans at dose K+2: 1-5e8 cells/kg | Experimental | Biological: gp91 GransAdult CGD patients without systemic infection will participate in a dose-escalation trial to identify the most effective yet safe dose of study agent. Subjects enrolled will receive 1 administration of study agent at dose K+2, and safety of dose will be determined. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (22 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| gp91 GransAdults with gp91phox-deficient CGD without systemic infection will participate in a dose escalation trial to identify the MTD (the most effective yet safe dose) of gp91 Grans IV infusion. | Biological | IV infusion of gp91-Grans at dose K+1:1e7 cells/kg, IV infusion of gp91-Grans at dose K+2: 1-5e8 cells/kg, IV infusion of gp91-Grans at dose K: 1e6 cells/kg |
Primary outcome
Feasibility: Recruitment, implementation, and manufacturing of gp91-Grans for infusions.
Time frame: 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 patients
Time frame: 3 months
Determine an MTD for administration.
Safety: Frequency of grade 3 or greater adverse events or serious adverse events related to the study agent
Time frame: 3 months
Determine safety of gp91-Grans infusion.
2 secondary outcomes
Determine percent of circulating dihydrorhodamine (DHR) positive granulocytes following study agent infusion.
Time frame: 3 months
Serial measurement of circulating DHR+ granulocytes from peripheral blood until day 3 following study agent infusion or disappearance of DHR+ granulocytes.
Time frame: 3 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- Background: PubMed 28342916Marciano BE, Allen ES, Conry-Cantilena C, Kristosturyan E, Klein HG, Fleisher TA, Holland SM, Malech HL, Rosenzweig SD. Granulocyte transfusions in patients with chronic granulomatous disease and refractory infections: The NIH experience. J Allergy Clin Immunol. 2017 Aug;140(2):622-625. doi: 10.1016/j.jaci.2017.02.026. Epub 2017 Mar 22.
- Background: PubMed 8937413Stroncek DF, Leonard K, Eiber G, Malech HL, Gallin JI, Leitman SF. Alloimmunization after granulocyte transfusions. Transfusion. 1996 Nov-Dec;36(11-12):1009-15. doi: 10.1046/j.1537-2995.1996.36111297091747.x.
- Background: PubMed 21175646Heim KF, Fleisher TA, Stroncek DF, Holland SM, Gallin JI, Malech HL, Leitman SF. The relationship between alloimmunization and posttransfusion granulocyte survival: experience in a chronic granulomatous disease cohort. Transfusion. 2011 Jun;51(6):1154-62. doi: 10.1111/j.1537-2995.2010.02993.x. Epub 2010 Dec 22.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Suk S De Ravin, M.D. principal investigator
National Institute of Allergy and Infectious Diseases (NIAID)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing gp91 Grans in people with Chronic Granulomatous Disease and Infection, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Joanna L Peterson ((301) 346-9780, joanna.peterson@nih.gov), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Feasibility: Recruitment, implementation, and manufacturing of gp91-Grans for infusions. (time frame: 3 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05189925, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Oct 2026.