Table 3.
Phase I and II trial design for the first-in-human, Food and Drug Administration-approved clinical trial utilizing NSI-566RSC in patients with amyotrophic lateral sclerosis
| Phase I trial design | |||||
| Group | Number of patients | Subject details | Injection target | Injection details | Final cell dose |
| A1 | 3 | Nonambulatory | Lumbar | 5 unilateral | 5 × 105 |
| A2 | 3 | Nonambulatory | Lumbar | 10 bilateral | 1 × 106 |
| B | 3 | Ambulatory | Lumbar | 5 unilateral | 5 × 105 |
| C/E | 3 | Ambulatory | Lumbar | 10 bilateral | 1 × 106 |
| Cervical | 5 unilateral | 5 × 105 | |||
| D | 3 | Ambulatory | Cervical | 5 unilateral | 5 × 105 |
| Phase II trial design | |||||
| Group | Number of patients | Injection target | Number of injections | Final cell dose | |
| A | 3 | Cervical | 10 bilateral | 2 × 106 | |
| B | 3 | Cervical | 20 bilateral | 4 × 106 | |
| C | 3 | Cervical | 20 bilateral | 6 × 106 | |
| D | 3 | Cervical | 20 bilateral | 8 × 106 | |
| E | 3 | Lumbar and cervical | 20 bilateral for each target | 16 × 106 | |