N Engl J Med

Does teplizumab prevent disease progression in type 1 diabetes?

October 24, 2023

Among children and adolescents with newly diagnosed type 1 diabetes, two 12-day courses of teplizumab were associated with preservation of β-cell function, but no significant differences between groups were observed with respect to secondary end points. 

• In the phase 3, randomized, placebo-controlled PROTECT trial, researchers assessed β-cell preservation, clinical end points, and safety in children and adolescents who were assigned to receive teplizumab or placebo for two 12-day courses. Primary end point: change from baseline in β-cell function, measured by stimulated C-peptide levels at week 78. Key secondary end points: insulin doses required to meet glycemic goals, HbA1c levels, time in target glucose range, and clinically important hypoglycemic events.
• Participants treated with teplizumab (n=217) had significantly higher stimulated C-peptide levels than patients receiving placebo (n=111) at week 78 (least-squares mean difference, 0.13 pmol/mL; 95% confidence interval [CI], 0.09 to 0.17; P<0.001); and 94.9% (95% CI, 89.5-97.6) of patients treated with teplizumab maintained a clinically meaningful peak C-peptide level of ≥0.2 pmol/mL, vs. 79.2% (95% CI, 67.7-87.4) of placebo recipients. The groups didn’t differ significantly in terms of key secondary end points.
• Adverse events occurred primarily in association with administration of study drug and included headache, GI symptoms, rash, lymphopenia, and mild cytokine release syndrome.

Source:

Ramos EL, et al. (2023, October 18). N Engl J Med.Teplizumab and β-Cell Function in Newly Diagnosed Type 1 Diabetes. https://pubmed.ncbi.nlm.nih.gov/37861217/