Autologous Mobilized Peripheral Blood CD34+ Hematopoietic Stem and Progenitor Cells (HSPC) Transduced With the Elongation Factor Alpha Short Promoter (EFS) - Adenosine Deaminase (ADA) Gene (EFS-ADA) Lentiviral Vector for Adenosine Deaminase Severe Combined Immune Deficiency (ADA SCID)

NCTID	NCT05432310 (View at clinicaltrials.gov)
Description	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints. (Show More)
Indication	Severe Combined Immunodeficiency, ADA-deficient
Compound Name	Simoladagene autotemcel (formerly OTL-101)
Sponsor	University of California, Los Angeles
Funder Type	Other
Status	Recruiting
Enrollment Count	20

Target Gene/Variant	ADA
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type
Dose 1	Transduced CD34+ cells, minimum dose: Fresh (1 x 10^6 cells/kg); Cryopreserved (2 x 10^6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
Recent Updates	Administered Under Compassionate Use