Safety and Dose-Escalation Trial of mRNA-Corrected Granulocyte Cells Infusion for Adult Males with Chronic Granulomatous Disease

What this Trial Is About

Researchers are evaluating a new procedure for males aged 18 to 75 with Chronic Granulomatous Disease CGD, a genetic condition where the bodys cells cannot effectively kill germs, leading to frequent or severe infections. This phase 1, open-label trial aims to test the safety and feasibility of giving patients granulocyte-enriched blood cells corrected with messenger RNA mRNA to restore the function of a critical protein called gp91phox. The study seeks to find the maximum tolerated dose of these corrected cells through a dose-escalation design. Participants will undergo a process called apheresis to collect their white blood cells, which will then be treated with mRNA to correct the gene mutation before being infused back into their bloodstream. The study includes three increasing dose levels of the corrected cells, each tested in groups of at least three patients. Participants will be hospitalized for at least seven days for the cell collection, infusion, and monitoring. Following discharge, they will keep a symptom diary, have weekly contact for one month, monthly follow-ups, and a final visit three months after the infusion. During the study, participants will have multiple assessments including physical exams, blood and urine tests, and throat swabs to monitor safety and immune responses. Researchers will measure the presence and activity of the corrected granulocytes in the blood, monitor for adverse events, and assess inflammatory and immune reactions. The total study participation lasts about three months after the infusion, with regular contact and evaluations to track the treatments effects and safety.

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