A Two-Period Open-label Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism

A Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism

The objective of the trial is to evaluate the efficacy and safety of dasiglucagon administered as a subcutaneous (SC) infusion in reducing hypoglycemia in children with CHI.

Inclusion Criteria:

• Established and documented diagnosis of CHI based on standard of care
• Experiencing ≥3 events of hypoglycemia per week (plasma glucose [PG] <70 mg/dL [<3.9 mmol/L]) according to the investigator's evaluation
• Previously undergone near-total pancreatectomy or being treated with a non-surgical approach, having been evaluated as not eligible for pancreatic surgery
• If somatostatin analogues or sirolimus are used, the therapy should be well established as judged by the investigator, especially when considering their biological half-life

Exclusion Criteria:

• Previous administration of dasiglucagon
• Known or suspected allergy to the trial drug or related products
• Previous participation (randomization) in this trial
• Circulatory instability requiring supportive medication or presence of pheochromocytoma
• Requires exogenous insulin
• Body weight of <4 kg (8.8 lbs.)
• Documented HbA1c ≥7% subsequent to near-total pancreatectomy and within 6 months prior to screening
• Known or suspected presence of significant central nervous system disease/injury such that in the investigator's opinion will affect trial participation
• Use of systemic corticosteroids, e.g., hydrocortisone >20 mg/m2 body surface area or equivalent in the 5 days before screening
• Use of anti-inflammatory biological agents, or other immune modulating agents in the 3 months prior to screening
• Any clinically significant abnormality identified on echocardiogram that in the opinion of the investigator would affect the patient's ability to participate in the trial
• Any recognized clotting or bleeding disorders
• Has participated in an interventional clinical trial (investigational or marketed product) within 3 months of screening or 5 half-lives of the drug under investigation (whichever comes first), or plans to participate in another clinical trial.