Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)

NCT01306019 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

RECRUITING

Status

40

Enrollment

NIH

Sponsor class

Conditions

X-linked Severe Combined Immunodeficiency (XSCID)

Interventions

BIOLOGICAL: Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector
DRUG: Busulfan
DRUG: Palifermin

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)