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

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).