Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism

NCT ID: NCT04172441

Last Updated: 2025-03-14

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-06-19
• Study Completion Date: 2022-03-07

Brief Summary

The objective of the trial is to evaluate the efficacy of dasiglucagon in reducing glucose requirements in children with persistent congenital hyperinsulinism (CHI) requiring continuous intravenous (IV) glucose administration to prevent/manage hypoglycemia.

Conditions

Congenital Hyperinsulinism

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

dasiglucagon first then placebo

48 hours of dasiglucagon subcutaneous (sc) infusion starting at 10 µg/hour with crossover to 48 hours placebo sc infusion (part 1) followed by 21 days of dasiglucagon sc infusion (part 2).

Group Type: EXPERIMENTAL

dasiglucagon

Intervention Type: DRUG

Glucagon analogue

Placebo

Intervention Type: DRUG

Placebo for dasiglucagon

placebo first then dasiglucagon

48 hours of placebo sc infusion with crossover to 48 hours dasiglucagon sc infusion starting at 10 µg/hour (part 1) followed by 21 days of dasiglucagon sc infusion (part 2).

Group Type: EXPERIMENTAL

dasiglucagon

Intervention Type: DRUG

Glucagon analogue

Placebo

Intervention Type: DRUG

Placebo for dasiglucagon

Interventions

dasiglucagon

Glucagon analogue

Intervention Type: DRUG

Placebo

Placebo for dasiglucagon

Intervention Type: DRUG

Other Intervention Names

ZP4207

Eligibility Criteria

Inclusion Criteria

• CHI diagnosis established based on the following:

1. Hyperinsulinemia: plasma insulin above the limit of detection of the assay documented during an event of hypoglycemia, and/or

2. Hypofattyacidemia: plasma free fatty acid <1.7 mmol/L, and/or

3. Hypoketonemia: Beta-hydroxybutyrate <1.8 mmol/L, and/or

4. Glycemic response: an increase in plasma glucose (PG) of >30 mg/dL (1.7 mmol/L) after 1 mg IV or intramuscular (IM) glucagon administration

• Male or female, age ≥7 days and <12 months at screening
• Body weight of ≥2.0 kg (4.4 lbs.)
• Continuous IV glucose requirement to prevent hypoglycemia

Exclusion Criteria

• Is suspected of having a transient form of CHI (e.g., transient hyperinsulinism due to maternal diabetes or perinatal stress)
• Was born preterm below 34 weeks of gestational age
• Presence of hypertension or hypotension, including circulatory instability requiring supportive medication or presence of pheochromocytoma
• Known or suspected presence of severe brain damage
• Evidence of metabolic, endocrine, or syndromic causes of hypoglycemia not due to hyperinsulinism
• Use of systemic corticosteroids, e.g., hydrocortisone >20 mg/m^2 body surface area or equivalent within 5 days before screening
• Prior use of lanreotide, sirolimus (mechanistic target of rapamycin [mTOR] inhibitors), anti-inflammatory biological agents, or other immune modulating agents. Prior use of octreotide is allowed after a minimum of 48 hour washout before randomization.
• Any clinically significant abnormality identified on echocardiogram that in the opinion of the investigator would affect the subject's ability to participate in the trial
• Any recognized clotting or bleeding disorder
• The use of prescription or non-prescription medications known to cause QT prolongation

• Minimum Eligible Age: 7 Days
• Maximum Eligible Age: 364 Days
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Zealand Pharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Jelena Ivkovic, MD

Role: STUDY_DIRECTOR

Zealand Pharma

Locations

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Cook Children's Medical Center

Fort Worth, Texas, United States

University Children's Hospital

Düsseldorf, , Germany

University Hospital, Magdeburg

Magdeburg, , Germany

Hadassah Medical Center

Jerusalem, , Israel

Manchester University NHS Foundation Trust

Manchester, , United Kingdom

Countries

United States; Germany; Israel; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2017-004545-24

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ZP4207-17103

Identifier Type: -

Identifier Source: org_study_id