Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene

NCT01852071 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

COMPLETED

Status

46

Enrollment

OTHER

Sponsor class

Conditions

ADA-SCID

Interventions

GENETIC: Infusion of autologous EFS-ADA LV CD34+ (OTL-101)
DRUG: busulfan
DRUG: PEG-ADA ERT

Sponsor

University of California, Los Angeles

Collaborators

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