Early Promise for New Agent in Congenital Hyperinsulinism in
A rare disorder, occurring in 1 in 20,000 to 1 in 50,000 US live births, congenital hyperinsulinism is caused by several different genetic mutations. Babies are diagnosed shortly after birth because they have severe hypoglycemia and require high glucose infusion rates to maintain normal glucose levels.

The current study involved 16 children (6 girls, 10 boys), aged 10 months through 15 years with persistent hypoglycemia due to congenital hyperinsulinism. There were four parts to the study: parts 1 and 2 investigated the effect on fasting glucose of three different dosing regimens of exendin-(9-39) delivered by infusion (group 1, 0.28 mg/kg; group 2, 0.44 mg/kg; group 3, 0.6 mg/kg) compared with vehicle. In parts 3 and 4, a subset of eight subjects (three girls, five boys) received either vehicle or 0.6 mg/kg exendin-(9-39) during a mixed-meal tolerance test and an oral protein tolerance test. The KATP channel mutation form of hyperinsulinism is characterized by both fasting- and protein-induced hypoglycemia.

During fasting and with adjustment for order of administration, the area under the curve of fasting plasma glucose was significantly higher in treatment group 2 compared with vehicle (P = .037). As a result, fasting hypoglycemia was reduced by 76% in group 2 (P = .009) and by 84% in group 3 (P = .014). Plasma insulin levels didn't change during the mixed-meal test (P = .827) but nearly doubled with exendin-(9-39) during the protein challenge test (P = .004). C-peptide didn't significantly change (P = .059).

Source: https://www.medscape.com/viewarticle/972526#vp_2