Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease

NCT06325709 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

RECRUITING

Status

10

Enrollment

NIH

Sponsor class

Conditions

Chronic Granulomatous Disease (CGD)
X-Linked Chronic Granulomatous Disease

Interventions

DRUG: Plerixafor
DRUG: Filgrastim
DRUG: Palifermin
DRUG: Busulfan
BIOLOGICAL: Base-edited hematopoietic stem and progenitor cells
DRUG: Sirolimus

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)