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

ZP4207-17109

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.

2019-02-07

2020-08-31

2020-10-05

Completed

Early phase I

32

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.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 32, 'type': 'ACTUAL'}}

2023-12-13