Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

NCT02999984 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

COMPLETED

Status

10

Enrollment

OTHER

Sponsor class

Conditions

Severe Combined Immunodeficiency Due to ADA Deficiency

Interventions

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

Sponsor

University of California, Los Angeles

Collaborators

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