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Low-Dose Transplant Drug Preserves Beta Cells in Young People With Type 1 Diabetes, Phase 2 Trial Finds

An adaptive trial in newly diagnosed patients identified 0.5 mg/kg as the minimum effective dose with fewer adverse events than a higher dose.

Overview

  • Researchers tested anti‑thymocyte globulin (ATG) in a multicentre, double‑blind, randomized, placebo‑controlled, adaptive dose‑ranging study across 14 hospitals in eight European countries.
  • ATG was infused over two consecutive days, with participants aged 5–25 years randomized to placebo, 0.5 mg/kg, or 2.5 mg/kg after lower and intermediate doses were dropped per protocol.
  • The primary endpoint—stimulated C‑peptide area under the curve at 12 months—showed that 0.5 mg/kg preserved beta‑cell function comparably to 2.5 mg/kg versus placebo.
  • Safety favored the lower dose: cytokine release syndrome occurred in 33% at 2.5 mg/kg versus 24% at 0.5 mg/kg, and serum sickness in 82% versus 32%, with none in the placebo group.
  • Investigators recommend 0.5 mg/kg for young patients, noting it can be given as a single‑day infusion and is widely available at affordable prices; results were published in The Lancet and presented at EASD.