NCT06559176View on ClinicalTrials.gov

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

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

12

Participants

Timeline

Start Date

October 17, 2024

Primary Completion Date

January 31, 2030

Study Completion Date

February 28, 2030

Conditions
Chronic Granulomatous DiseaseGranulomatous Disease, Chronic
Interventions
BIOLOGICAL

PM359

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

Trial Locations (5)

20892

NIH Clinical Center, Bethesda

37203

The Children's Hospital at Tristar Medical Group/Sarah Cannon Center for Blood Cancers, Nashville

90027

University of California Los Angeles Medical Center, Los Angeles

H3T 1C5

CHU - Sainte Justine Hospital, Montreal

NW1 2PG

University College of London Hospital, London

Sponsors
All Listed Sponsors
lead

Prime Medicine, Inc.

INDUSTRY

NCT06559176 - A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD ) | Biotech Hunter | Biotech Hunter