SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection

NCTID	NCT03217617 (View at clinicaltrials.gov)
Description	This is a Phase I/II gene therapy trial treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector (ivlv-X1) to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous lentiviral gene transfer protocol. (Show More)
Development Status	Active
Indication	X-linked Severe Combined Immunodeficiency (XSCID)
Disease Ontology Term	DOID:0060013
Compound Name	Ivlv-X1 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	IL2RG
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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