The efficacy and safety of the dipeptidyl peptidase-4 inhibitor saxagliptin in treatment-naive patients with type 2 diabetes mellitus: a randomized controlled trial

Background: The aim of this study was to assess efficacy and safety of saxagliptin monotherapy for up to 76 weeks in patients with type 2 diabetes mellitus (T2DM) and inadequate glycemic control, with main efficacy assessment at 24 weeks. Methods: 365 treatment-naive patients with T2DM (HbA(1c) 7.0%-10.0%) were treated with saxagliptin 2.5 mg q.A.M., saxagliptin 2.5 mg q.A.M. with possible titration to saxagliptin 5 mg, saxagliptin 5 mg q.A.M., saxagliptin 5 mg q.P. M., or placebo. After week 24, patients in all groups were eligible for titration to saxagliptin 10 mg based on HbA(1c) >= 7%, and all unrescued placebo patients began blinded metformin 500 mg/day. Rescue with open-label metformin was available for patients with inadequate glycemic control. Results: At week 24, placebo-subtracted mean HbA(1c) reduction from baseline (LOCF) was significantly greater in the saxagliptin treatment groups vs placebo, and remained greater through week 76. Serious adverse events (AEs) and discontinuations due to AEs were similar in saxagliptin and control groups; incidence of confirmed hypoglycemia was low across all treatment groups (saxagliptin-treated, 2 [0.7]; control, 1 [1.4]). Conclusions: In treatment-naive patients with T2DM, saxagliptin monotherapy demonstrated statistically significant improvement in HbA(1c) compared with placebo at 24 weeks and was generally well tolerated for up to 76 weeks. Trial registration: ClinicalTrials.gov Identifier: NCT00316082