Ustekinumab for type 1 diabetes in adolescents: a multicenter, double-blind, randomized phase 2 trial
Authors' conclusion
Although exploratory, our data suggest a role for an activated subset of TH17.1 cells in T1D that can be targeted with minimal adverse effects to reduce C-peptide loss, which requires confirmation in a larger study.
Study context
- Population
- Adolescents aged 12–18 years with recent-onset T1D
- Participants
- 72 adolescents
- Comparison
- Ustekinumab vs control
- Primary endpoint
- β-cell function, measured by stimulated C-peptide
- Follow-up
- 12 months
Methodological detailsdouble-blind, multicenter, phase 2
- Double-blind
- Multicenter
- Phase 2
Primary result
As reported in the abstract
Stimulated C-peptide (β-cell function)
- β-cell function 49% higher in intervention group, meeting prespecified primary outcome
- P = 0.02
Further findings
Correlates of C-peptide preservation (exploratory)
- C-peptide preservation correlated with reduction of TH17.1 cells
- P = 0.04
- C-peptide preservation correlated with reduction in IL-2+ GM-CSF+ TH17.1 cells
- P = 0.04
- Significant fall in proinsulin-specific IL-17A-secreting T cells
- P = 0.0003
- Exploratory
Safety
- Well tolerated with no increase in adverse events
Original publication
Ustekinumab for type 1 diabetes in adolescents: a multicenter, double-blind, randomized phase 2 trial. Nat Med. Published 30 July 2024. doi:10.
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