High dose insulin given orally to children at risk of developing type 1 diabetes resulted in an immune response without hypoglycaemia, a double blind, placebo controlled pilot study reports.

According to the authors the findings support the need for a phase III trial to determine whether oral insulin can prevent islet autoimmunity and diabetes in genetically at risk children.

The Pre-POINT study included 25 children aged 2 to 7 with a strong family history of type 1 diabetes who were autoantibody negative. They were randomly assigned to receive oral insulin or placebo daily once for 3 to 18 months.

Nine children received insulin with dose escalations from 2.5 to 7.5 mg (n = 3), 2.5 to 22.5 mg (n = 3), or 7.5 to 67.5 mg (n = 3) after 6 months; 6 children received doses of 22.5 mg (n = 3) or 67.5 mg (n = 3).

Immune responses to insulin were seen in 2 of 10 children (20%) treated with placebo, in 1 of 6 children (16.7%) treated with 2.5 mg of insulin, 1 of 6 (16.7%) treated with 7.5 mg, 2 of 6 (33.3%) treated with 22.5 mg, and 5 of 6 (83.3%) treated with 67.5 mg, the German researchers reported in JAMA.

Adverse events did not differ between groups and hypoglycaemia was not observed at any of the tested doses.

The results “increases enthusiasm for cautiously moving forward with a study of primary prevention in genetically screened children,” an accompanying editorial said.