ZP4207-17103, 2017-004545-24, phase 2 clinical trial in Hyperinsulinism

This is a combined phase 2 and 3, randomised, double-blind, parallel-group trial to evaluate the efficacy and safety of dasiglucagon in children aged = 7 days and < 3 months at screening who have been diagnosed with diffuse or focal CHI. To qualify for participation, patients must have an absolute necessity during the last 24 hours of intravenous (IV) glucose (=10 mg/kg/min) to prevent hypoglycaemia and be non-responsive to oral diazoxide (defined as inadequacy of diazoxide to eliminate the need for IV glucose, not necessarily that that diazoxide has no effect).
The purpose of this trial is to evaluate whether subcutaneous pump infusion of dasiglucagon can effectively and safely reduce glucose requirements sufficiently to enable discharge of patients from hospital care.
After screening, eligible patients will be randomly assigned in a double-blind fashion to receive dasiglucagon or placebo for 24 hours (Treatment Period 1), see Figure 1. During this period, dosing of trial drug as well as the glucose infusion rate (GIR) will be adjusted based on blood glucose measurements, and feeding (full feedings by mouth, nasogastric tube, or gastric tube) should remain as stable as possible. Safety assessments will be performed 24 hours after initiation of the trial drug (active or placebo).