. 2014 Jan 16;8(1):e2613. doi: 10.1371/journal.pntd.0002613

Table 2. Interim analyses and non-comparative efficacy analysis for primary (day 210) and secondary (day 30) end points.

	Multiple dose			Single dose			Single dose
	7×3 mg/kg			7.5 mg/kg			10 mg/kg
Parasite clearance at day 30 (IC)	No of patients(N)	No. cured(n) (%)	95% CI^*	N	n (%)	95% CI^*	N	n (%)	95% CI^*
Interim analysis 1^‡	18	16 (89)	65–99	20	10 (50)	27–73	-	-	-
Interim analysis 2^**	25	19 (74)	55–91	-	-	-	20	16 (80)	56–94
Interim analysis 3^§	44	37 (84)	70–93	-	-	-	40	29 (73)	56–85
Cure at Day 30
Overall	62^φ	53 (85)	74–93	20	10 (50)	27–73	40	29 (73)	56–85
Kassab, Sudan	18	16 (89)	65–99	-	-	-	18	13 (72)	47–90
Gondar, Ethiopia	20	13 (65)	41–85	9	2(22)	3–60	9	3 (33)	7–70
Arba Minch, Ethiopia	24	24 (100)	86–100^†	11	8 (73)	39–94	13	13 (100)	75–100^†
Cure at Day 210 (6 months follow-up)
Overall	54^φ	46 (85)	73–93	20	8 (40)	19–64	40	23 (58)	41–73
Kassab, Sudan	17	13 (76)	50–93	-	-	-	18	7 (39)	17–64
Gondar, Ethiopia	14	10 (71)	42–92	9	1 (11)	<1–48	9	3 (33)	7–70
Arba Minch, Ethiopia	23	23 (100)	83–100^†	11	7 (64)	31–89	13	13 (100)	75–100^†

Intention-to-Treat and Per-Protocol complete-case analysis populations were identical at day 30 & day 210.

Exact binomial 95% confidence interval (CI).

One-sided 97.5% confidence interval.

Multiple versus 7.5 mg/kg single dose; p-value = 0.015 (Fisher's exact test): Dose escalation rule met; increase dosage to 10 mg/kg & continue recruitment.

Multiple versus 10 mg/kg single dose; p value = 0.748 from chi-square test of difference between arms: Dose escalation rule not met; continue recruitment (same dosage in single-dose arm).

Includes patients in Interim analysis 2. Multiple versus 10 mg/kg single dose; p-value = 0.196 from chi-square test of difference between arms: Dose escalation rule not met; concerns arose regarding low cure in each arm and recruitment not continued.

8 patients lost to follow-up by day 210 (all from the multiple-dose arm).