Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
Gene Therapy Via Intravenous Injection of Lentiviral Vector (Ivlv-ADA) for the Treatment of Adenosine Deaminase-severe Combined Immunodeficiency (ADA-SCID)
Shenzhen Geno-Immune Medical Institute
10 participants
Jun 30, 2024
INTERVENTIONAL
Conditions
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.
Eligibility
Inclusion Criteria9
- Diagnosis of classical ADA-SCID based on:
- A proven defective adenosine deaminase (ADA) gene as defined by direct sequencing of patient DNA.
- T-cell immune deficiency defined as one or more of the following: CD3+ autologous T cells \< 300/ul, or less than 50% of normal value for in vitro mitogen stimulation, or absent proliferation in vitro to antigens.
- With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus or fungus; disseminated BCG infection.
- No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia of children.
- No prior allogeneic stem cell transplantation.
- Life expectancy ≥ 2 months.
- Negative for HIV infection.
- Written, informed consent obtained prior to any study-specific procedures.
Exclusion Criteria1
- None
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Interventions
Injection of ivlv-ADA lentiviral vector at \~1x10\^9 per kg body weight
Locations(2)
View Full Details on ClinicalTrials.gov
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NCT03645460