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NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)

NCT05189925 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2026-04-24

No results posted yet for this study

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.

Conditions

Interventions

BIOLOGICAL

gp91 Grans

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.

Sponsors & Collaborators

  • National Institute of Allergy and Infectious Diseases (NIAID)

    lead NIH

Principal Investigators

  • Suk S De Ravin, M.D. · National Institute of Allergy and Infectious Diseases (NIAID)

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-07-22
Primary Completion
2026-07-01
Completion
2026-07-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05189925 on ClinicalTrials.gov