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Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
Sponsor: Shenzhen Geno-Immune Medical Institute
Summary
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.
Official title: Gene Therapy Via Intravenous Injection of Lentiviral Vector (Ivlv-ADA) for the Treatment of Adenosine Deaminase-severe Combined Immunodeficiency (ADA-SCID)
Key Details
Gender
All
Age Range
1 Month - Any
Study Type
INTERVENTIONAL
Enrollment
10
Start Date
2024-06-30
Completion Date
2027-12-31
Last Updated
2025-09-09
Healthy Volunteers
No
Interventions
Direct intravenous injection of ivlv-ADA lentiviral vector
Injection of ivlv-ADA lentiviral vector at \~1x10\^9 per kg body weight
Locations (2)
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China
Guilin Hospital of Chinese Traditional and Western Medicine
Guilin, Guangxi, China