Table 3.
Percentages of adaptive trial simulations that terminate for efficacy / futility by analysis stage
| Simulations (%) that end for efficacy by subgroup | Total % of simulations that end for efficacy |
Total % of simulations that end for futility |
Total % of simulations that end |
|||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Analysis stage at study completion |
C1 | C1–2 | C1–3 | C1–4 | C1–5 | C1–6 | ||||
| Cat #1 | 1st interim (n=450) | 3.0% | 0.0% | 0.0% | 0.2% | 0.3% | 3.9% | 7.4% | 8.4% | 15.8% |
| 2nd interim (n=675) | 4.4% | 0.0% | 0.0% | 0.2% | 0.5% | 5.6% | 10.8% | 12.1% | 22.9% | |
| Final (n=900) | 11.8% | 0.1% | 0.1% | 0.6% | 1.2% | 15.1% | 28.9% | 32.5%** | 61.4% | |
| Total | 19.2% | 0.1% | 0.2% | 1.0% | 2.0% | 24.6% | 47.0%* | 53.0% | 100.0% | |
| Cat #2 | 1st interim (n=450) | 0.1% | 7.5% | 0.1% | 0.0% | 0.1% | 5.2% | 12.9% | 2.7% | 15.6% |
| 2nd interim (n=675) | 0.1% | 14.5% | 0.2% | 0.0% | 0.2% | 10.0% | 24.9% | 5.3% | 30.2% | |
| Final (n=900) | 0.2% | 25.9% | 0.3% | 0.0% | 0.4% | 17.9% | 44.7% | 9.5%** | 54.2% | |
| Total | 0.4% | 47.8% | 0.6% | 0.0% | 0.7% | 33.0% | 82.5%* | 17.5% | 100.0% | |
Cat #1 indicates categorization scheme #1 in which patients are categorized based on time and location of the arterial occlusion. Cat #2 indicates categorization scheme #2 in which patients are categorized only based on time. C1-y indicates a subgroup that consists of consecutive patient categories. See figure 1 for the exact criteria that define each patient category in scheme #1 and #2. C1–6 includes all 6 patient categories and thus indicates that the trial completes without subgroup selection
The overall total corresponds to the power of the adaptive trial design
The percentage at the final analysis (n=900) reflects the number of simulations that neither stopped for efficacy or futility at an interim analysis nor demonstrated efficacy at the final analysis.