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Completed

NCT Number: NCT04172441

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

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.

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Key information

Conditions

Congenital Hyperinsulinism Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Glucose Metabolism Disorders Hyperinsulinism Hypoglycemia Infant, Newborn, Diseases Metabolic Diseases Nutritional and Metabolic Diseases Pancreatic Diseases

Age range

7 day–364 day

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

University Children's Hospital, Düsseldorf, Germany

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • CHI diagnosis established based on the following:
  • Hyperinsulinemia: plasma insulin above the limit of detection of the assay documented during an event of hypoglycemia, and/or
  • Hypofattyacidemia: plasma free fatty acid <1.7 mmol/L, and/or
  • Hypoketonemia: Beta-hydroxybutyrate <1.8 mmol/L, and/or
  • 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

Treatment and study plan

Dasiglucagon

Drug

Glucagon analogue

Other names: ZP4207

Placebo

Drug

Placebo for dasiglucagon

Primary outcomes

  1. Mean Intravenous Glucose Infusion Rate

    Time frame: Hours 36-48 after initiation of trial drug (Part 1)

    Mean intravenous (IV) glucose infusion rate (GIR) in the last 12 hours of each treatment period during Part 1, the crossover part of the trial (dasiglucagon or placebo administration).

Secondary outcomes

  1. Carbohydrates Administered

    Time frame: 0 to 48 hours after initiation of trial drug

    Total amount (g) of carbohydrates administered during the crossover part of the trial (dasiglucagon or placebo administration) per day.

  2. Mean Intravenous Glucose Infusion Rate

    Time frame: 48 hours after initiation of trial drug (Part 1)

    Mean IV GIR for each 48-hour treatment period during Part 1, the crossover part of the trial (dasiglucagon or placebo administration).

  3. Mean Intravenous Glucose Infusion Rate Below 10 mg/kg/Minute

    Time frame: Hours 36-48 after initiation of trial drug (Part 1)

    Mean IV GIR below 10 mg/kg/minute in the last 12 hours of each treatment period during Part 1, the crossover part of the trial (dasiglucagon or placebo administration) (yes/no)

  4. Time to Complete Weaning Off Intravenous Glucose

    Time frame: Days 5 to 25 (Part 2)

    Time in days to complete weaning off IV glucose administration during Part 2, defined as the first point in time when the patient had been off IV glucose administration for at least 12 hours.

  5. Hypoglycemia Event Rate in Part 2

    Time frame: Days 5 to 25

    Hypoglycemia event rate, defined as number of hypoglycemic events when PG was <70 mg/dL (or 3.9 mmol/L), as detected by self-monitored plasma glucose.

  6. Clinically Significant Hypoglycemia Events in Part 2

    Time frame: Days 5 to 25

    Clinically significant hypoglycemia event rate, defined as number of events when PG was <54 mg/dL (3.0 mmol/L), as detected by self-monitored plasma glucose.

  7. Time to Actual Hospital Discharge

    Time frame: Days 5 to 25

    Time in days to actual hospital discharge defined as the time from first exposure to the study drug in Part 2 to discharge from hospital.

  8. Time to Pancreatic Surgery

    Time frame: Days 5 to 25

    Time (days) to pancreatic surgery (sub-total or total pancreatectomy with a cutoff of ≥95%).

  9. Carbohydrates Administered

    Time frame: Days 5 to 25

    Total amount (g) of carbohydrates administered (regardless of the route) per day.

  10. Carbohydrates Administered Intravenously

    Time frame: Days 5 to 25

    Amount (g) of carbohydrates administered via IV glucose infusion or bolus or total parenteral nutrition. This secondary endpoint was intended to account only for carbohydrates administered via IV glucose infusion or bolus. It was not possible to differentiate between carbohydrates administered via IV glucose infusion or bolus and carbohydrates administered as being, or not being, part of total parenteral nutrition from the collected data. This endpoint was expanded to include both.

  11. Carbohydrates Administered Parenterally

    Time frame: Days 5 to 25

    Amount (g) of carbohydrates administered as part of total parenteral nutrition.

  12. Carbohydrates Administered Orally

    Time frame: Days 5 to 25

    Amount (g) of carbohydrates administered via oral route.

  13. Carbohydrates Administered Via Gastric Feed

    Time frame: Days 5 to 25

    Amount (g) of carbohydrates administered via nasogastric tube or gastrostomy.

  14. Time in Range in Part 2

    Time frame: Days 5 to 25

    Percent time in range (PG between 70 to 180 mg/dL [3.9-10.0 mmol/L]) as measured by continuous glucose monitoring.

  15. Time in Hypoglycemia in Part 2

    Time frame: Days 5 to 25

    Percent time in hypoglycemia (when PG was <70 mg/dL [or 3.9 mmol/L]) as measured by continuous glucose monitoring.

  16. Time in Clinically Significant Hypoglycemia in Part 2

    Time frame: Days 5 to 25

    Percent time in clinically significant hypoglycemia (when PG was <54 mg/dL [or 3.0 mmol/L]) as measured by continuous glucose monitoring.

  17. Hypoglycemia Episodes in Part 2

    Time frame: Days 5 to 25

    Rate of hypoglycemia episodes, defined as number of episodes per week when PG was <70 mg/dL (3.9 mmol/L) for 15 minutes or more, as measured by continuous glucose monitoring.

  18. Clinically Significant Hypoglycemia Episodes in Part 2

    Time frame: Days 5 to 25

    Rate of clinically significant hypoglycemia episodes, defined as number of episodes per week when was <54 mg/dL (3.0 mmol/L) for 15 minutes or more, as measured by continuous glucose monitoring.

  19. Extent of Hypoglycemia in Part 2

    Time frame: Days 5 to 25

    Extent of hypoglycemia (defined as the area over the glucose curve [AOCglucose] below 70 mg/dL [3.9 mmol/L]) as measured by continuous glucose monitoring.

  20. Extent of Clinically Significant Hypoglycemia in Part 2

    Time frame: Days 5 to 25

    Extent of clinically significant hypoglycemia (defined as the area over the glucose curve [AOCglucose] below 54 mg/dL [3.0 mmol/L]) as measured by continuous glucose monitoring, divided by the total duration in hours of continuous glucose monitoring.

  21. Time in Hyperglycemia in Part 2

    Time frame: Days 5 to 25

    Percent time in hyperglycemia (when PG was >180 mg/dL [10.0 mmol/L]), as measured by continuous glucose monitoring.

Sponsors and collaborators

Lead sponsor

Zealand Pharma

Industry

Registry information

Official study title

A Randomized Trial in 2 Parts: Double-Blind, Placebo-Controlled, Crossover Part 1 and Open-label Part 2, Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism

Important dates

Study start
2020
Primary completion
2022
Study completion
2022
First posted
Nov 21, 2019
Registry last updated
Mar 14, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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