SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) Via Direct Intravenous Injection of Lentiviral Vector (Ivlv-X1)
Shenzhen Geno-Immune Medical Institute
10 participants
Jun 30, 2024
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria10
- Diagnosis of SCID-X1 based on:
- A proven mutation in the common interleukin-2 receptor gamma chain 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 antigen stimulation.
- No available HLA identical related donor.
- With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus; disseminated BCG infection.
- No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia.
- No prior allogeneic stem cell transplantation.
- Life expectancy ≥ 3 months.
- Documented to be negative for HIV infection.
- Written, informed consent obtained prior to any study-specific procedures.
Exclusion Criteria8
- No available molecular diagnosis confirming SCID-X1.
- Existence of an available HLA-identical related donor.
- Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease.
- Current treatment with any chemotherapeutic agent (becomes eligible if not on treatment for at least 1 month).
- Patients with evidence of infection with HIV-1 or 2.
- Presence of a medical condition indicating that survival will be less than 4 weeks such as the requirement for mechanical ventilation, severe failure of a major organ system, or evidence of a serious, progressive infection that is refractory to medical treatment.
- Current treatment with any immunosuppressive agent, excluding corticosteroids.
- Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
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Interventions
ivlv-X1 LV intravenous injection at a dose of \~1x10e9/kg body weight.
Locations(2)
View Full Details on ClinicalTrials.gov
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NCT03217617