Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)

NCTID	NCT03645460 (View at clinicaltrials.gov)
Description	This is a Phase I/II trial of in vivo lentiviral gene therapy for treating adenosine deaminase severe combined immunodeficiency (ADA-SCID) using a self-inactivating lentiviral vector (LV) ivlv-ADA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous (iv) LV gene therapy protocol. (Show More)
Development Status	Active
Indication	Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Disease Ontology Term	DOID:5810
Compound Name	ADA lentiviral vector
Sponsor	Shenzhen Geno-Immune Medical Institute
Funder Type	Other
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	10
Results Posted	Not Available

Target Gene/Variant	ADA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	LV
Editor Type
Dose 1	1E9 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	China

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