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 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
Enrollment Count
10
Results Posted
Not Available
Therapy Information
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
Study Record Dates
Current Stage
Na
Submit Date
2018-07-17
Completion Date
2027-12-31
Last Update
2024-07-17
Participation Criteria
Eligible Age
>=1 Month
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
2
Locations
China
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Resources/Links
Resources/Links
No External Links Available.