Clinical Trial

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

Enrolling by Invitation Phase 1/2
View on ClinicalTrials.gov →
Summary
This is an open-label, single-arm, multicenter Phase 1/2 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34+ stem cells modified ex vivo (PM359) in participants with autosomal recessive Chronic Granulomatous Disease (CGD) caused by mutations in the NCF1 (Neutrophil Cytosolic Factor 1) gene.
Protocol Amendment History 12 amendments
This ClinicalTrials.gov record has been amended 12 times since 2024-08-16; most recent amendment 2026-03-31.
Status change: Active, Not Recruiting → Enrolling by Invitation 2026-03-31
Status change: Recruiting → Active, Not Recruiting 2025-05-21
Status change: Not Yet Recruiting → Recruiting 2024-09-25
Trial Details
NCT Number NCT06559176
Lead Sponsor Prime Medicine, Inc.
Conditions Chronic Granulomatous Disease, Granulomatous Disease, Chronic
Enrollment 12 participants
Start Date 2024-10-17
Primary Completion 2030-01 (estimated)
Study Completion 2030-02 (estimated)
Updated on ClinicalTrials.gov 2026-04-06