NCT03217617

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)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Shenzhen Geno-Immune Medical Institute · Academic / Other
• Sex: Male
• Age: 1 Month – 1 Year
• Healthy volunteers: Not accepted

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.

Detailed description

X-linked severe combined immunodeficiency (SCID-X1) is a genetic disorder caused by defect in the common cytokine receptor chain, normally on the surface of lymphocytes. Individuals with SCID-X1 lack the normal development of a functional immune system and so have difficulty fighting infections, which may lead to chronic or severe illness and death. X-SCID patients are normally rescued by a bone marrow transplant from a healthy donor. This trial aims to treat SCID-X1 using a self-inactivating lentiviral vector (LV) carrying a functional gene to correct the genetic defect. By direct intravenous (iv) injection of the LV (ivlv-X1) to modify immune cells in the body, this treatment may establish normal healthy immune cells and overcome the immunodeficiency. The primary objectives are to evaluate the safety of the self-inactivating ivlv-X1-LV, the in vivo gene transfer protocol and the efficacy of immune reconstitution in patients overcoming frequent infections present at the time of treatment, assessment of iv LV gene transfer efficiency, and finally the long-term correction of immunodeficiency.

Conditions

• SCID, X-Linked

Interventions

Type	Name	Description
BIOLOGICAL	Direct intravenous injection of ivlv-X1 lentiviral vector	ivlv-X1 LV intravenous injection at a dose of \~1x10e9/kg body weight.

Timeline

• Start date: 2024-06-30
• Primary completion: 2026-12-31
• Completion: 2027-12-31
• First posted: 2017-07-14
• Last updated: 2025-09-09

Locations

2 sites across 1 country: China