Efficacy and Safety of New Orodispersible Tablet Formulations of Praziquantel (Racemate and L-Praziquantel) in Schistosoma mansoni-Infected Preschool-Age Children and Infants: A Randomized Dose-Finding Phase 2 Study

Introduction Two novel formulations of praziquantel (PZQ) and Levo-(L-)PZQ (arpraziquantel) have been developed for the treatment schistosomiasis in preschool-age children infants. Methods This open-label, dose-finding Phase 2 study assessed efficacy safety PZQ infants infected with Schisostoma mansoni Côte d’Ivoire. In Part 1, 420 aged 2.1–6.9 years (weight 10.0–29.9 kg) were enrolled randomized to one 7 arms (n=60 per arm): commercially available racemate (rac)-PZQ at 3x20 mg/kg or 40 (treatment 1 2); rac-PZQ orodispersible tablet (ODT) 60 3 4); L-PZQ ODT 30 mg/kg, 45 5, 6, 7). The optimal formulation dose identified (L-PZQ 50 mg/kg) was used 2, which 24 6–24 months 7.5–14.8 kg). Infants treated an age-staggered approach: age 13–24 arm 8, n=20) 6–12 9, n=4). primary endpoint clinical cure rate (CR) demonstrated by Kato–Katz method 14–21 days post-treatment. Secondary endpoints included CR point-of-care circulating cathodic antigen, egg reduction (ERR), adverse events (AEs). Results CRs ≥70% achieved all highest (89.7%), (89.5%), (86.0%). >90%. All had ERRs >95%. Treatment-related AEs reported 71 participants (16.0%) similar across arms; most mild transient. common treatment-emergent laboratory abnormalities. No deaths discontinuation due no new concerns identified. Conclusion New as single-dose therapy against S. acceptable overall infants, warranting further studies this population. Clinical Trial Registration ClinicalTrials.gov , identifier NCT02806232 ; Pan African Trials Registry, PACTR201604001493593 .