Table 1.
Ongoing clinical trials using lentiviral vectors to modify hematopoietic stem cells
| Condition | Phase | NCT number |
|---|---|---|
| Transfusion-dependent β-thalassemia | 1/2 | NCT02453477 |
| 3 | NCT02906202 | |
| Cerebral adrenoleukodystrophy | 2/3 | NCT01896102 |
| Sickle cell disease | 1 | NCT02140554 |
| 1 | NCT02193191 | |
| Metachromatic leukodystrophy and adrenoleukodystrophy | 1/2 | NCT02559830 |
| Wiskott-Aldrich syndrome | 1/2 | NCT01347346 |
| 1/2 | NCT01347242 | |
| 1/2 | NCT02333760 | |
| X-SCID | 1/2 | NCT01306019 |
| 1/2 | NCT01512888 | |
| ADA-SCID | 1/2 | NCT02999984 |
| 1/2 | NCT01380990 | |
| Fanconi anemia | 2 | NCT02931071 |
| X-linked chronic granulomatous disease | 1/2 | NCT02234934 |
ADA adenosine deaminase, SCID severe combined immunodeficiency