Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
Investigational drug(s) / intervention(s)
Direct intravenous injection of ivlv-ADA lentiviral vector
Direct intravenous injection of ivlv-ADA lentiviral vector: Injection of ivlv-ADA lentiviral vector at \~1x10\^9 per kg body weight
Study 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
Sex
ALL
Min age
1 Month
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* 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 Criteria:
* None
Primary outcome measure(s)
Overall survival up to a year — 1 years Patient will be monitored for overall health condition, including immune cell assessments, blood biochemistry and metabolitic activities, metabolic detoxification, gene-modified cell percentage and vector copy number (VCN) in the blood, and continued follow-up for 1 years.
Trial sites (1)
Facility
City
Region
Status
Shenzhen Geno-immune Medical Institute
Shenzhen
Guangdong
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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