Efficacy and Safety of Luseogliflozin (TS-071) in Japanese Children and Adolescents with Type 2 Diabetes Mellitus: A Multicenter, Randomized, Double-masked, Placebo-controlled Phase 3 Study.

INTRODUCTION: This study evaluated the efficacy and safety of luseogliflozin (TS-071), a sodium-glucose cotransporter 2 (SGLT2) inhibitor, in Japanese children and adolescents with type 2 diabetes (T2D). METHODS: In this multicenter, randomized, double-masked, placebo-controlled phase 3 trial, patients aged 10-17 years with hemoglobin A1c (HbA1c) levels of 7.0%-12.0%, with or without prior metformin use, were enrolled. Participants were randomized (2:1) to receive luseogliflozin 2.5 mg or placebo for 12 weeks (period I), followed by a 40-week open-label extension (period II), allowing dose escalation to 5 mg. RESULTS: Forty-eight participants were randomized and received luseogliflozin (n = 31) or placebo (n = 17). The primary endpoint-change in HbA1c from baseline at the end of period I-was -1.45% (95% CI, -1.78% to -1.11%) in the luseogliflozin group. The between-group difference in HbA1c change was -1.76% (95% CI, -2.49% to -1.02%). Luseogliflozin maintained efficacy through 52 weeks, with a mean HbA1c change of -0.92% ± 1.74% at the end of period II. Adverse events (AEs) and adverse drug reactions (ADRs) occurred in 85.4% and 29.2% of patients, respectively. No deaths occurred; three serious AEs and one mild hypoglycemic event were reported. Urinary and genital infections were the most frequent ADRs. CONCLUSION: Luseogliflozin significantly improved glycemic control and was well tolerated, supporting its potential as an effective and safe treatment option for children and adolescents with T2D.