Table 4.

Planning Parameters and Sample Sizes for Recent Onset Trials (Statistical Power is 85% for all calculations)

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).

^ΔMinimal 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.