Table 1.
Phase and status of gene therapy clinical trials.
| Phase I | Phase I/II | Phase II | Phase II/III | Phase III | Phase IV | Single subject | Total | |
|---|---|---|---|---|---|---|---|---|
| Open | 970 | 327 | 288 | 15 | 71 | 2 | 5 | 1,678 |
| (57.81%) | (19.49%) | (17.16%) | (0.89%) | (4.23%) | (0.12%) | (0.30%) | (71.86%) | |
| Closed | 337 | 129 | 96 | 7 | 11 | – | – | 580 |
| (58.10%) | (22.24%) | (16.55%) | (1.21%) | (1.90%) | (24.83%) | |||
| Withdrawn | 12 | 8 | 8 | – | – | – | – | 28 |
| (42.86%) | (28.57%) | (28.57%) | (1.20%) | |||||
| On clinical hold | 3 | 3 | 1 | – | – | – | – | 7 |
| (42.86%) | (42.86%) | (14.28%) | (0.30%) | |||||
| Conditional approval | 9 | 1 | 5 | – | – | – | – | 15 |
| (60.00%) | (6.67%) | (33.33%) | (0.64%) | |||||
| Canceled | – | 2 | 1 | 1 | 2 | – | – | 6 |
| (33.33%) | (16.67%) | (16.67%) | (33.33%) | (0.26%) | ||||
| Under review | 10 | 3 | 1 | – | 1 | – | – | 15 |
| (66.67%) | (20.00%) | (6.67%) | (6.67%) | (0.64%) | ||||
| Submission not | 2 | 1 | 2 | – | 1 | – | – | 6 |
| completed | (33.33%) | (16.67%) | (33.33%) | (16.67%) | (0.26%) | |||
| Total | 1,343 | 474 | 402 | 23 | 86 | 2 | 5 | 2,335 |
| (57.52%) | (20.30%) | (17.21%) | (0.99%) | (3.68%) | (0.09%) | (0.21%) | (100.00%) |