Actively Recruiting
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
Led by Shenzhen Geno-Immune Medical Institute · Updated on 2025-09-09
10
Participants Needed
2
Research Sites
182 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of classical ADA-SCID confirmed by defective ADA gene sequencing
- T-cell immune deficiency shown by CD3+ T cells < 300/ul, or less than 50% normal response to mitogen stimulation, or absent antigen proliferation
- Presence of severe infections such as pneumonitis, prolonged diarrhea needing nutrition support, herpes virus, adenovirus, fungal infections, or disseminated BCG infection
- No cytogenetic abnormalities or leukemia-associated rearrangements
- No prior allogeneic stem cell transplantation
- Life expectancy of at least 2 months
- Negative HIV test
- Written informed consent obtained before study procedures
You will not qualify if you...
- None
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China, 518000
Actively Recruiting
2
Guilin Hospital of Chinese Traditional and Western Medicine
Guilin, Guangxi, China
Actively Recruiting
Research Team
L
Lung-Ji Chang, Ph.D
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here