Gene Therapy for X Linked Severe Combined Immunodeficiency

NCTID	NCT04286815 (View at clinicaltrials.gov)
Description	A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency ）.The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number per cell) in blood and bone marrow cells, immune reconstitution vector insertion-site patterns and so on. (Show More)
Development Status	Active
Indication	Severe Combined Immunodeficiency, X-linked
Disease Ontology Term	DOID:0060013
Compound Name	Lentiviral vector/IL2RG
Sponsor	Children's Hospital of Chongqing Medical University
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	Undisclosed
Mechanism of Action	Functional gene replacement
Route of Administration	Undisclosed
Drug Product Type	Undisclosed
Target Tissue/Cell
Delivery System	Undisclosed
Vector Type	undisclosed
Editor Type
Dose 1	Undisclosed
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

No External Links Available.