Table 4.
No. of Treatment Arms | Planning Parameters (from Table 3) | Other Planning Parameters | Total Sample Size | |||||
---|---|---|---|---|---|---|---|---|
Estimates Used* | Standard Deviation (RMSE) | Predicted Control Group Mean | MDDΔ | α level† (sides of test) | Allocation Ratio (C:E)• | Excluding Those with Missing Endpoint | Including Those Missing Endpoint (inflated 10%) | |
2 | RMSE & PCGM | 0.152 | 0.334 | 50 | 0.05 (1) | 1:2 | 15+29 | 16+32 |
90% LB of PCGM | 0.152 | 0.319 | 50 | 0.05 (1) | 1:2 | 16+31 | 17+34 | |
90% UB of RMSE | 0.158 | 0.334 | 50 | 0.05 (1) | 1:2 | 16+31 | 17+34 | |
Both LB of PCGM & UB RMSE | 0.158 | 0.319 | 50 | 0.05 (1) | 1:2 | 17+33 | 19+37 | |
Unadjusted Estimates (t-test) | 0.232 | 0.332 | 50 | 0.05 (1) | 1:2 | 34+67 | 37+74 | |
5 studies combined estimates | 0.152 | 0.334 | 65 | 0.05 (1) | 1:2 | 9+18 | 10+20 | |
90% LB of control group mean | 0.152 | 0.319 | 65 | 0.05 (1) | 1:2 | 10+19 | 11+21 | |
90% UB of RMSE | 0.158 | 0.334 | 65 | 0.05 (1) | 1:2 | 10+19 | 11+21 | |
Both LB mean & UB RMSE | 0.158 | 0.319 | 65 | 0.05 (1) | 1:2 | 11+21 | 12+23 | |
Unadjusted Estimates (t-test) | 0.232 | 0.332 | 65 | 0.05 (1) | 1:2 | 21+41 | 23+46 | |
5 studies combined estimates | 0.152 | 0.334 | 50 | 0.05 (2) | 1:2 | 18+36 | 20+40 | |
90% LB of control group mean | 0.152 | 0.319 | 50 | 0.05 (2) | 1:2 | 19+38 | 22+43 | |
90% UB of RMSE | 0.158 | 0.334 | 50 | 0.05 (2) | 1:2 | 20+39 | 22+43 | |
Both LB mean & UB RMSE | 0.158 | 0.319 | 50 | 0.05 (2) | 1:2 | 21+42 | 23+46 | |
Unadjusted Estimates (t-test) | 0.232 | 0.332 | 50 | 0.05 (2) | 1:2 | 42+84 | 47+93 | |
5 studies combined estimates | 0.152 | 0.334 | 65 | 0.05 (2) | 1:2 | 11+22 | 13+25 | |
90% LB of control group mean | 0.152 | 0.319 | 65 | 0.05 (2) | 1:2 | 12+24 | 13+26 | |
90% UB of RMSE | 0.158 | 0.334 | 65 | 0.05 (2) | 1:2 | 12+24 | 14+27 | |
Both LB mean & UB RMSE | 0.158 | 0.319 | 65 | 0.05 (2) | 1:2 | 13+26 | 14+28 | |
Unadjusted Estimates (t-test) | 0.232 | 0.332 | 65 | 0.05 (2) | 1:2 | 26+52 | 29+57 | |
3 | RMSE & PCGM | 0.152 | 0.334 | 50 | 0.025 (1) | 1:1:1 | 3×24=72 | 3×27=81 |
90% LB of PCGM | 0.152 | 0.319 | 50 | 0.025 (1) | 1:1:1 | 3×26=78 | 3×29=87 | |
90% UB of RMSE | 0.158 | 0.334 | 50 | 0.025 (1) | 1:1:1 | 3×26=78 | 3×29=87 | |
Both LB of PCGM & UB RMSE | 0.158 | 0.319 | 50 | 0.025 (1) | 1:1:1 | 3×28=84 | 3×31=93 | |
Unadjusted Estimates (t-test) | 0.232 | 0.332 | 50 | 0.025 (1) | 1:1:1 | 3×56=168 | 3×62=186 | |
5 studies combined estimates | 0.152 | 0.334 | 65 | 0.025 (1) | 1:1:1 | 3×15=45 | 3×17=51 | |
90% LB of control group mean | 0.152 | 0.319 | 65 | 0.025 (1) | 1:1:1 | 3×16=48 | 3×18=54 | |
90% UB of RMSE | 0.158 | 0.334 | 65 | 0.025 (1) | 1:1:1 | 3×16=48 | 3×18=54 | |
Both LB mean & UB RMSE | 0.158 | 0.319 | 65 | 0.025 (1) | 1:1:1 | 3×17=51 | 3×19=57 | |
Unadjusted Estimates (t-test) | 0.232 | 0.332 | 65 | 0.025 (1) | 1:1:1 | 3×35=105 | 3×38=114 |
All estimates are based on the five studies combined. All 4 combinations of the estimate or 90% upper/lower bound of the estimate for both RMSE and the predicted control group mean are listed. Along with, the observed control group mean and the pooled unbiased estimate of the standard deviation (pooled over treatment group).
Minimal Detectable Difference expressed as a percent increase on the original C-Peptide scale of the experimental group mean to the predicted control group mean.
For the 3 treatment arm trials two-pairwise tests will be performed, thereby controlling the overall type I error at 0.05
C is the Control Group and E is the Experimental Group.