Effect of heat shock protein peptide DiaPep277 on β-cell function in paediatric and adult patients with recent-onset diabetes mellitus type 1:: two prospective, randomized, double-blind phase II trials

Background Aim of this trial was to test whether heat shock protein peptide DiaPep277 treatment in adult and paediatric patients with recent-onset type 1 diabetes (T1D) is safe and whether it can preserve endogenous insulin production. Methods Two studies were performed in a prospective, multicentre, double-blind, placebo-controlled trial. Fifty adult (study p520, aged 16-44 years) and 49 paediatric patients (study p521, 4-15 years) with recent-onset T1D were treated subcutaneously at four different time points with 0.2 mg or 1.0 mg DiaPep277 versus placebo and followed for 18 months. Adult patients were treated with 0.2 mg, 1.0 mg or 2.5 mg DiaPep277 versus placebo. Stimulated C-peptide served as readout for functional beta-cell-mass. Results DiaPep277-treatment was not associated with severe side effects. No differences were found in placebo and DiaPep277 treated groups. In adults, a modest trend towards better maintenance of beta-cell function was observed in the 0.2 mg and 1.0 mg group, while there was significant loss of stimulated C-peptide in the placebo and 2.5 Ing group. Paediatric patients with low HLA risk showed stable C-peptide levels until 13 months upon treatment with 1 mg DiaPep277. Despite similar stimulated C-peptide levels at baseline, children exhibited a more pronounced loss of beta-cell function over 18 months than adults (p = 0.0003). Conclusion Administration of DiaPep277 seems safe and may have beneficial effects on C-peptide levels over time in some patients with TID, but this finding was not accompanied by reduced HbA(1c) or insulin requirement. Studies with more patients and longer follow-up are needed to further study the effect of DiaPep277. Copyright (C) 2006 John Wiley & Sons, Ltd.