SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
China10 participantsStarted 2024-06-30
Plain-language 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.
Who can participate
Age range1 Month – 1 Year
SexMALE
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Inclusion criteria
✓. Diagnosis of SCID-X1 based on:
✓. 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 criteria
✕. 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.