Efficacy and safety of low-dose interleukin 2 for Behçet's syndrome: a randomized, placebo-controlled, double-blind, phase 2 clinical trial.
- 2026-01-07
- Nature communications 17(1)
- PubMed: 41501036
- DOI: 10.1038/s41467-025-68100-w
Study Design
- Type
- Randomized Controlled Trial (RCT)
- Sample size
- n = 60
- Population
- 60 participants with active oral ulcers due to Behçet's syndrome
- Methods
- randomized, double-blind, placebo-controlled trial, low-dose interleukin-2 (LD-IL-2) or placebo
- Blinding
- Double-blind
- Duration
- 12 weeks
- Rigorous Journal
Behçet's syndrome (BS), a chronic vasculitis, leads to recurrent oral ulcers, severely impacting quality of life. Here we report the results of a randomized, double-blind, placebo-controlled trial (NCT04065672) to evaluate the efficacy and safety of low-dose interleukin-2 (LD-IL-2) in BS patients. 60 participants with active oral ulcers are randomly assigned to receive LD-IL-2 or placebo. The primary endpoint is the oral ulcer count at week 12. The LD-IL-2 group has significantly fewer oral ulcers than the placebo group (0.69 ± 1.05 vs. 1.57 ± 0.90, P = 0.001) with greater improvements in ulcer pain, disease activity, and quality of life. No infections or severe adverse events are observed in either group. LD-IL-2 expands regulatory T (Treg) cells and decreased the ratio of effector T cell to Treg cells. Thus, LD-IL-2 therapy might be an effective and safe treatment in BS patients and is associated with the modulation of CD4 + T cell populations.