Early Metabolic Endpoints Identify Persistent Treatment Efficacy in Recent-Onset Type 1 Diabetes Immunotherapy Trials
Authors' conclusion
Early dynamic measures can identify a treatment effect among successful immune therapies in type 1 diabetes trials with good long-term prediction and practical sample size over a 6-month period. While external validation of these findings is required, strong rationale and data exist in support of shortening early-phase clinical trials.
Study context
- Population
- Participants with recent-onset T1D in six TrialNet immunotherapy RCTs6 RCTs
- Comparison
- Immunotherapy vs control in TrialNet trials; early (3- and 6-month) dynamic metabolic measures vs 12-month C-peptide AUC
- Primary endpoint
- Treatment effect on mixed-meal tolerance test-stimulated C-peptide AUC as a dynamic measure (change from baseline) at early time points, and prediction of 12-month C-peptide AUC
- Follow-up
- 3 and 6 months
Methodological detailspooled and individual-trial analysis, partitioned into successful and unsuccessful trials
- Pooled and individual-trial analysis
- Partitioned into successful and unsuccessful trials
Primary result
As reported in the abstract
Treatment effect on C-peptide AUC change from baseline, trials meeting their primary end point
- Treatment effect at 3 months (successful trials)
- P = 0.030
- Treatment effect at 6 months (successful trials)
- P < 0.001
Further findings
Prediction of 12-month C-peptide AUC by 6-month dynamic C-peptide AUC (adjusted for age and baseline value)
- R2
- 0.80
- P
- P < 0.001
- Supported smaller trial sizes down to
- 54 participants
Original publication
Early Metabolic Endpoints Identify Persistent Treatment Efficacy in Recent-Onset Type 1 Diabetes Immunotherapy Trials. Diabetes Care. Published 15 April 2024. doi:10.
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