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

NCT06559176 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-04-06

No results posted yet for this study

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.

Conditions

  • Chronic Granulomatous Disease
  • Granulomatous Disease, Chronic

Interventions

BIOLOGICAL

PM359

Single dose of PM359 administered autologously by intravenous (I.V.) infusion following myeloablative conditioning with busulfan

Sponsors & Collaborators

  • Prime Medicine, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-10-17
Primary Completion
2030-01-31
Completion
2030-02-28
FDA Drug
Yes

Countries

  • United States
  • Canada
  • United Kingdom

Study Locations

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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 NCT06559176 on ClinicalTrials.gov