Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study

NCT06959771 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

RECRUITING

Status

1

Enrollment

NIH

Sponsor class

Conditions

CD40L-HyperIgM Syndrome

Interventions

BIOLOGICAL: Base-edited hematopoietic stem and progenitor cells
DRUG: Alemtuzumab
DRUG: Sirolimus
DRUG: Palifermin
DRUG: Busulfan
BIOLOGICAL: Base-edited T lymphocyte cells

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)