Efficacy and safety of tirzepatide in children and adolescents with type 2 diabetes (SURPASS-PEDS): a randomised, double-blind, placebo-controlled, phase 3 trial.
Summary
In a 39-site, eight-country, double-blind phase 3 RCT with 99 adolescents, tirzepatide significantly improved glycemic control and reduced BMI versus placebo over 30 weeks, with effects sustained through a 22-week open-label extension to 1 year. Participants had youth-onset type 2 diabetes inadequately controlled on metformin and/or basal insulin.
Key Findings
- Multinational, double-blind phase 3 RCT across 39 sites in eight countries randomized 99 participants aged 10 to <18 years.
- Tirzepatide significantly improved glycemic control versus placebo at 30 weeks, with BMI reductions observed.
- Benefits in glycemic control and BMI were sustained through a 22-week open-label extension to 1 year.
Clinical Implications
Tirzepatide could expand treatment options for adolescents with type 2 diabetes, supporting earlier intensification when metformin and/or basal insulin are insufficient. Careful long-term safety monitoring in growth and puberty is warranted.
Why It Matters
This is the first multinational phase 3 randomized trial of tirzepatide in youth-onset type 2 diabetes, addressing a major therapeutic gap with clinically meaningful improvements in HbA1c and BMI.
Limitations
- Modest sample size (n=99) limits precision for safety and subgroup analyses
- Double-blind period was 30 weeks; longer-term blinded efficacy and safety data are needed
- Industry-funded study may introduce sponsorship bias
Future Directions
Head-to-head trials versus GLP-1 receptor agonists, assessment of durability and safety across puberty, and real-world effectiveness studies in diverse pediatric populations.
Study Information
- Study Type
- RCT
- Research Domain
- Treatment
- Evidence Level
- I - High-quality randomized, double-blind, placebo-controlled phase 3 trial
- Study Design
- OTHER