Fig. 1.

The standard study design for proof-of-concept trials of novel interventions in new-onset T1D. This is a randomized, placebo-controlled, double-blind phase 2 design, with 2:1 randomization (drug to placebo). Key inclusion criteria are shown. The primary endpoint, generally at 1 year, is the change from baseline in C-peptide area under the curve (AUC) following a mixed-meal tolerance test (MMTT). Secondary endpoints and continued safety follow-up usually extend to 2 years. Reprinted with permission from Ehlers & Nepom [10].