Actively Recruiting
SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
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 gene therapy trial treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector (ivlv-X1) to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous lentiviral gene transfer protocol.
CONDITIONS
Official Title
SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of SCID-X1 confirmed by a mutation in the interleukin-2 receptor gamma chain gene and T-cell immune deficiency as defined by CD3+ autologous T cells < 300/ul, less than 50% normal in vitro mitogen stimulation, or absent antigen response
- No available HLA identical related donor
- Presence of severe infections such as pneumonitis, protracted diarrhea requiring total parenteral nutrition, herpes virus or adenovirus infection, or disseminated BCG infection
- No cytogenetic abnormalities or acute leukemia-associated rearrangements
- No prior allogeneic stem cell transplantation
- Life expectancy of at least 3 months
- Documented negative HIV infection
- Written informed consent obtained before any study procedures
You will not qualify if you...
- No molecular diagnosis confirming SCID-X1
- Availability of an HLA-identical related donor
- Active malignant disease except EBV-associated lymphoproliferative disease
- Current chemotherapy treatment (unless stopped for at least 1 month)
- Evidence of HIV-1 or HIV-2 infection
- Medical conditions indicating survival less than 4 weeks, including need for mechanical ventilation, severe organ failure, or refractory serious infection
- Current treatment with immunosuppressive agents other than corticosteroids
- Investigator's judgment that patient is not eligible or unable to comply with study requirements
AI-Screening
AI-Powered Screening
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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
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