drugset / Trial / NCT03645460
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2024-06-30
- Primary completion
- 2026-12-31
- Completion
- 2027-12-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ivlv-ADA | Unknown | 1e+09 vg/kg | Intravenous |