Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy

NCT06851767 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

ENROLLING_BY_INVITATION

Status

18

Enrollment

NIH

Sponsor class

Conditions

X-linked Severe Combined Immunodeficiency
X-SCID
XSCID

Interventions

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

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)