Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency

NCT03315078 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

UNKNOWN

Status

13

Enrollment

NIH

Sponsor class

Conditions

X-Linked Combined Immunodeficiency Diseases

Interventions

BIOLOGICAL: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT
DRUG: Palifermin
DRUG: Busulfan

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)