Heat‐shock protein peptide DiaPep277 treatment in children with newly diagnosed type 1 diabetes: a randomised, double‐blind phase II study

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Abstract
Background Type 1 diabetes mellitus (T1DM) is a T‐cell‐mediated autoimmune disease that leads to the destruction of insulin‐producing beta cells. Treatment with DiaPep277, a peptide derived from heat‐shock protein 60 (hsp60), has been found to slow the deterioration of beta‐cell function after clinical onset of diabetes in NOD mice and human adults. Our aim was to evaluate the efficacy and safety of DiaPep277 treatment in attenuating beta‐cell destruction in children with recent‐onset T1DM. Methods A prospective, randomized, double‐blind, phase II design was used. The sample included 30 children (19 males) aged 7–14 years who had been diagnosed with T1DM from 53 to 116 days previously, and had basal C‐peptide concentrations above 0.1 nmol/L. The children were randomized to receive subcutaneous injections of 1 mg DiaPep277 (15 patients) or 40 mg mannitol (placebo) at entry and at 1, 6, and 12 months. The duration of follow‐up was 18 months. The groups were compared for stimulated C‐peptide level, exogenous insulin dose, and HbA1c concentration. Results C‐peptide levels similarly decreased over time in the DiaPep277‐ and placebo‐treated patients. There was no significant difference in insulin dose or HbA1c concentration between the groups at any time point. No serious drug‐related adverse effects were recorded throughout the study period. Conclusions One‐year treatment with DiaPep277 at a dosage of 1 mg is safe for use and well tolerated in children with recent‐onset T1DM. However, it appears to have no beneficial effect in preserving beta‐cell function or improving metabolic control. Copyright © 2007 John Wiley & Sons, Ltd.