The Journal of clinical endocrinology and metabolism · 2025
Dasiglucagon in Children With Congenital Hyperinsulinism Up to 1 Year of Age: Results From a Randomized Clinical Trial.
СтатьяРандомизированное исследованиеМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
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De Leon Diva D, Banerjee Indraneel, Kummer Sebastian, Birch Sune, Bøge Eva, Ivkovic Jelena, Kendall David M, Thornton Paul S
Аннотация
CONTEXT: Congenital hyperinsulinism (CHI) is a cause of persistent hypoglycemia in childhood with a considerable risk of lifelong neurological sequelae. Available pharmacological therapies are limited. Dasiglucagon is a glucagon analog for the treatment of hypoglycemia.
OBJECTIVE: To assess the efficacy and safety of dasiglucagon in children with CHI up to 1 year of age.
METHODS: This study included a randomized, crossover, double-blind, placebo-controlled part 1 and an open-label, single-arm part 2 at 4 centers in Germany, the United Kingdom, and the United States. Participants comprised children with CHI aged 7 days to 12 months who were dependent on IV glucose. In part 1, participants were randomized to dasiglucagon or placebo for 48 hours, then crossed over to the other treatment for 48 hours. In part 2, all participants received dasiglucagon for 21 days. The primary outcome was mean IV glucose infusion rate (GIR) in the last 12 hours of part 1.
RESULTS: Between June 19, 2020, and February 9, 2022, 12 eligible participants were randomized to dasiglucagon-placebo (n = 7) or placebo-dasiglucagon (n = 5). The IV GIR was significantly reduced with dasiglucagon compared with placebo (least-squares mean 4.3 mg/kg/min [95% confidence interval [CI], 1.04 to 7.60 mg/kg/min] and 9.5 mg/kg/min [95% CI, 6.24 to 12.81 mg/kg/min], respectively; P = .004). The most frequent adverse events in both treatment groups were gastrointestinal, dermatological, and metabolism and nutritional disorders.
CONCLUSION: In infants with CHI, dasiglucagon significantly reduced the amount of IV glucose needed to maintain euglycemia compared with placebo. Dasiglucagon represents a promising treatment for the management of CHI.
OBJECTIVE: To assess the efficacy and safety of dasiglucagon in children with CHI up to 1 year of age.
METHODS: This study included a randomized, crossover, double-blind, placebo-controlled part 1 and an open-label, single-arm part 2 at 4 centers in Germany, the United Kingdom, and the United States. Participants comprised children with CHI aged 7 days to 12 months who were dependent on IV glucose. In part 1, participants were randomized to dasiglucagon or placebo for 48 hours, then crossed over to the other treatment for 48 hours. In part 2, all participants received dasiglucagon for 21 days. The primary outcome was mean IV glucose infusion rate (GIR) in the last 12 hours of part 1.
RESULTS: Between June 19, 2020, and February 9, 2022, 12 eligible participants were randomized to dasiglucagon-placebo (n = 7) or placebo-dasiglucagon (n = 5). The IV GIR was significantly reduced with dasiglucagon compared with placebo (least-squares mean 4.3 mg/kg/min [95% confidence interval [CI], 1.04 to 7.60 mg/kg/min] and 9.5 mg/kg/min [95% CI, 6.24 to 12.81 mg/kg/min], respectively; P = .004). The most frequent adverse events in both treatment groups were gastrointestinal, dermatological, and metabolism and nutritional disorders.
CONCLUSION: In infants with CHI, dasiglucagon significantly reduced the amount of IV glucose needed to maintain euglycemia compared with placebo. Dasiglucagon represents a promising treatment for the management of CHI.