Breakthrough Phase III Trial Confirms Canagliflozin's Safety and Effectiveness for Treating Type 2 Diabetes in Youths

A comprehensive Phase III international clinical trial has demonstrated that canagliflozin is a safe and effective option for managing type 2 diabetes mellitus (T2DM) in children and adolescents. The study involved 171 participants between the ages of 10 and 18, who had inadequate glycemic control despite previous treatments. Conducted across 104 sites in 10 countries, the trial aimed to evaluate the drug's efficacy and safety profile in this young population.

Participants were randomly assigned to receive either 100 mg of oral canagliflozin daily or a matching placebo. Their blood sugar levels, specifically HbA1c and estimated glomerular filtration rate (eGFR), were monitored at week 12, with some participants undergoing dose adjustments based on their week 12 readings. The treatment continued for 52 weeks, with primary outcomes focusing on changes in HbA1c at week 26, and secondary measures including HbA1c at week 52, fasting plasma glucose, and body weight.

Results showed that those on canagliflozin experienced significantly greater reductions in HbA1c compared to placebo, with early improvements observed as soon as week six that persisted throughout the study. By week 52, a higher percentage of children treated with canagliflozin achieved target HbA1c levels below 6.5% and 7.0%. Fasting plasma glucose levels also improved markedly.

The safety profile of canagliflozin was comparable to that seen in adult trials. The most common adverse events included headaches, nasopharyngitis, urinary tract infections, and vomiting. Importantly, the incidence of hypoglycemia was low, and no severe hypoglycemic episodes were reported, supporting the drug’s safety in pediatric use.

These findings, published in the Annals of Internal Medicine, support expanding treatment options for young patients with T2DM, a group with limited effective therapies. The study emphasizes that canagliflozin is a promising addition to the current treatment landscape, providing robust glycemic control and a favorable safety profile.

Source: https://medicalxpress.com/news/2025-08-phase-iii-canagliflozin-diabetes-children.html