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

Zealand Pharma

Status and phase: Completed; Phase 3

Conditions: Congenital Hyperinsulinism

Treatments: Other: Standard of Care; Drug: Dasiglucagon

Study type: Interventional

Funder types: Industry

Identifiers

NCT03777176

ZP4207-17109

Details and patient eligibility

About

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.

Enrollment: 32 patients

Sex: All

Ages: 3 months to 12 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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32 participants in 2 patient groups

Standard of Care + dasiglucagon

Experimental group

Description:

4 weeks (Treatment Period 1) + 4 weeks (Treatment Period 2) of dasiglucagon treatment as an SC infusion starting at 10 µg/hr on top of standard of care

Treatment:

Drug: Dasiglucagon

Other: Standard of Care

Standard of Care Only

Other group

Description:

4 weeks (Treatment Period 1) of standard of care only + 4 weeks (Treatment Period 2) of dasiglucagon treatment as an SC infusion starting at 10 µg/hr on top of standard of care

Treatment:

Drug: Dasiglucagon

Other: Standard of Care