NCT06559176

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

A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Modified Ex Vivo Using Prime Editing (PM359) in Participants With Autosomal Recessive Chronic Granulomatous Disease Due to Mutations in the NCF1 Gene

• Status: Enrolling By Invitation
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Prime Medicine, Inc. · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Chronic Granulomatous Disease (CGD) is a rare genetic disease affecting the white blood cells, leading to failure of innate immunity against a variety of human pathogens and is also associated with autoimmune and inflammatory conditions. Approximately 20-25% of people with CGD inherit a mutation commonly known as "delGT" in both copies of the NCF1 gene, which encodes the p47phox protein. This study seeks to understand the safety and efficacy of a new gene editing technology, known as Prime Editing, in participants with autosomal recessive CGD caused by the delGT mutation in NCF1. Autologous CD34+ cells are collected from the participant via mobilization and apheresis, shipped to a central manufacturing facility and modified using Prime Editing to 'correct' the delGT mutation causing p47phox CGD. After manufacture, the Prime Edited stem cells (PM359) will be shipped to the study site, where they will be infused back into the participant following a preparative procedure known as conditioning. The study will initially enroll adult participants (aged ≥ 18) and plans to then move into adolescents aged 12 - 17, followed by children aged 6 - 11. The study is currently enrolling a limited number of participants by invitation. Treating physicians or patients may contact [e-mail] to inquire about potential participation.

Conditions

• Chronic Granulomatous Disease
• Granulomatous Disease, Chronic

Interventions

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

Timeline

• Start date: 2024-10-17
• Primary completion: 2030-01-01
• Completion: 2030-02-01
• First posted: 2024-08-19
• Last updated: 2026-04-06

Locations

5 sites across 3 countries: United States, Canada, United Kingdom

Regulatory

• FDA-regulated drug study