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mRNA Therapy

gp91 Grans for Chronic Granulomatous Disease

Phase 1

Recruiting

Led By Suk S De Ravin, M.D.

Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

CGD confirmed by DHR and gp91phox-deficiency subtype confirmed by protein analysis and/or genetic sequencing

Males aged 18 to 75 years

Must not have

Evidence of moderate to severe systemic infections as defined by fevers >=39°C within 3 days of treatment, ANC >12,000/microliter at screening, standard clinical diagnosis of pneumonia, liver abscess, or other deep tissue abscess, positive blood culture within 2 weeks of treatment, receipt of high-dose steroid within 30 days of screening, current or history of stage 4 chronic kidney disease, unstable diabetes mellitus, current or history of heart failure stage D, history of symptomatic arrhythmias, current or history of invasive cancers requiring chemotherapy within 5 years of screening, evidence of urinary tract infection, evidence of streptococcal pharyngitis, active hepatitis B, C, or HIV infections, unstable hypertension requiring addition of new anti-hypertensives within 2 weeks of screening, impaired renal function with serum creatinine >3.0 mg/dL, serum transaminases and bilirubin >3 x the upper limit of normal, electrocardiogram abnormalities indicative of acute myocardial injury or anesthetic risks, anemia with hemoglobin <8 g/dL, thrombocytopenia, profound thrombocytopenia not reversible with platelet transfusions, abnormal PT/PTT values outside accepted ranges, inherited bleeding disorder precluding line placement, severe oxygen-dependent pulmonary disease, history of alcohol or illicit drug abuse or dependence, participation in a clinical protocol that may affect study results

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 3 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new procedure to see if it can help people with a certain gene mutation kill germs better.

Who is the study for?

This trial is for males aged 18-75 with Chronic Granulomatous Disease (CGD) due to a gp91phox gene mutation. Participants must be able to consent, have follow-up care arranged, and agree to use contraception if they can cause pregnancy.

What is being tested?

The study tests a procedure where mRNA is added to blood cells in CGD patients. It aims to correct the gene defect temporarily so cells can better fight infections. Patients will undergo cell collection, receive mRNA-corrected cells via IV, and be monitored.

What are the potential side effects?

Potential side effects are not explicitly listed but may include reactions related to IV infusion such as discomfort at the injection site or an immune response against the modified cells.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My CGD and its gp91phox-deficiency subtype are confirmed by specific tests.

Select...

I am a man between 18 and 75 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~3 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Feasibility: Recruitment, implementation, and manufacturing of gp91-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

Safety: Frequency of grade 3 or greater adverse events or serious adverse events related to the study agent

Secondary study objectives

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.

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: IV infusion of gp91-Grans at dose K: 1e6 cells/kgExperimental Treatment1 Intervention

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.

Group II: IV infusion of gp91-Grans at dose K+2: 1-5e8 cells/kgExperimental Treatment1 Intervention

Group III: IV infusion of gp91-Grans at dose K+1:1e7 cells/kgExperimental Treatment1 Intervention

0 / 3Eligibility criteria met