SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection

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.

Timeline

Start

2024-06-30

Primary completion

2026-12-31

Completion

2027-12-31

Drugs

Indications