Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
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
Enrollment Count
10
Results Posted
Not Available
Therapy Information
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
Study Record Dates
Current Stage
Na
Submit Date
2020-02-23
Completion Date
2025-05-01
Last Update
2020-03-27
Participation Criteria
Eligible Age
<=18 Years
Standard Ages
Child, Adult
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
1
Locations
China
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Resources/Links
Resources/Links
No External Links Available.