Table 2.
HSC-GT clinical trials recruiting LSDs patients or currently active (LV: lentiviral vector).
| Disease | NCT Ref. | Intervention | Status | Est. Participants |
|---|---|---|---|---|
| MLD | NCT01560182 | OTL-200 (autologous CD34+ enriched cell fraction transduced with human ARSA LV) | Phase 1/2; Active, not recruiting | 20 |
| MLD | NCT03392987 | OTL-200 | Phase 2; Active, not recruiting | 10 |
| MLD | NCT04283227 | OTL-200 | Phase 3; Recruiting | 6 |
| MLD | NCT02559830 | Transduced CD34+ autologous HSCs | Phase 1/2; Recruiting | 50 |
| MPS IIIA | NCT04201405 | Autologous CD34+ cells transduced with the human SGSH LV | Phase 1/2; Recruiting | 5 |
| MPS I-H | NCT03488394 | Autologous CD34+ cells transduced with human IDUA LV | Phase 1/2; Recruiting | 8 |
| Cystinosis | NCT03897361 | CTNS-RD-04 (autologous CD34+ enriched cells transduced with human CTNS LV) | Phase 1/2; Recruiting | 6 |
| Fabry | NCT03454893 | AVR-RD-01 (autologous CD34+ cell-enriched fraction transduced with human AGA LV) | Phase 1/2; Recruiting | 12 |
| Gaucher | NCT04145037 | AVR-RD-02 (autologous CD34+ enriched HSCs transduced with human GBA gene LV) | Phase 1/2; Recruiting | 16 |