Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon in Children With Congenital Hyperinsulinism

An Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism

This is an open-label, multinational, multicenter, long-term safety and efficacy extension trial in patients with Congenital Hyperinsulinism (CHI) who completed either ZP4207-17103 or ZP4207-17109 (defined as lead-in trials).

The primary objective is to evaluate the long-term safety of dasiglucagon administered as subcutaneous (SC) infusion in children with CHI.

Study Overview

• Status: Active, not recruiting
• Conditions: Congenital Hyperinsulinism
• Intervention / Treatment: Drug: dasiglucagon

• Study Type: Interventional
• Enrollment (Estimated): 44
• Phase: Phase 3

Contacts and Locations

Study Locations

• Germany
  • Düsseldorf, Germany, 40225
    • University Hospital Düsseldorf, Department of Pediatrics
  • Magdeburg, Germany, 39120
    • Otto von Guericke University Magdeburg, Department of Pediatrics
• Israel
  • Jerusalem, Israel, 9765422
    • Hadassah Medical Center
• United Kingdom
  • Glasgow, United Kingdom
    • NHS Greater Glasgow and Clyde
  • Liverpool, United Kingdom
    • Alder Hey Children's Hospital NHS Foundation Trust
  • London, United Kingdom
    • Great Osmond Street Hospital for Children NHS Foundation Trust
  • Manchester, United Kingdom
    • Central Manchester University Hospital NHS Foundation Trust
• United States
  • Colorado
    • Aurora, Colorado, United States, 13123
      • Children's Hospital of Colorado
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month to 13 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Completed treatment in either Trial ZP4207-17103 or ZP4207-17109
• Expected to continue to have a positive benefit-risk assessment for treatment with dasiglucagon (based on considerations of glycemic effect, tolerability, and nature and frequency of adverse events experienced in the lead-in trial)

Exclusion Criteria:

• The patient developed any conditions prohibited by the lead-in trial, requires medication prohibited by the lead-in trial, or has other new complications that preclude participation in the investigator's opinion.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dasiglucagon open-label; Dasiglucagon treatment as sc infusion starting at 10 µg/hr on top of standard of care	Drug: dasiglucagon; Glucagon analog; Other Names: ZP4207

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events	Number of adverse events occurring up to Month 1, Month 1 to Month 3 and in each 3-month period for the first year; subsequent years will have longer periods assigned for analysis	Baseline through treatment completion, up to 3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Amount of gastric carbohydrates administered to treat hypoglycemia	Total amount of gastric carbohydrates administered via nasogastric tube or gastrostomy per week to treat hypoglycemia	Baseline through treatment completion, up to 3 years
Nasogastric (NG) tube or gastrostomy removal	Time to removal of NG tube or gastrostomy	Baseline through treatment completion, up to 3 years
Pancreatic surgery	Time to pancreatic surgery (sub-total or total pancreatectomy)	Baseline through treatment completion, up to 3 years
Time in hypoglycemia	Continuous glucose monitoring (CGM) percent time <70 mg/dL (3.9 mmol/L)	Baseline through treatment completion, up to 3 years
Hypoglycemia episodes	Rate of CGM-detected hypoglycemia episodes <70 mg/dL (3.9 mmol/L) for 15 minutes or more	Baseline through treatment completion, up to 3 years
Clinically significant episodes of hypoglycemia	Rate of clinically significant CGM-detected hypoglycemia episodes <54 mg/dL (3.0 mmol/L) for 15 minutes or more	Baseline through treatment completion, up to 3 years
Gastric carbohydrate administrations	Number of gastric carbohydrate administrations (nasogastric tube or gastrostomy) to treat hypoglycemia	Baseline through treatment completion, up to 3 years
Nightly gastric carbohydrate administrations	Number of nightly (midnight to 6 am) gastric carbohydrate administrations (nasogastric tube or gastrostomy) to treat hypoglycemia	Baseline through treatment completion, up to 3 years
Extent of hypoglycemia	Extent of hypoglycemia (area over the glucose curve [AOCglucose] below 70 mg/dL [3.9 mmol/L]) as measured by continous glucose monitoring (CGM)	Baseline through treatment completion, up to 3 years
Extent of clinically significant hypoglycemia	Extent of hypoglycemia (area over the glucose curve [AOCglucose] below 54 mg/dL [3.0 mmol/L]) as measured by continous glucose monitoring (CGM)	Baseline through treatment completion, up to 3 years
Diazoxide dose	Reduction in diazoxide dose in mg/kg body weight/day from start of lead-in trial	Baseline through treatment completion, up to 3 years
Somatostatin analog dose	Reduction in somatostatin analog dose from start of lead-in trial	Baseline through treatment completion, up to 3 years
Prescribed amount of continuous gastric carbohydrate administration	Change in total amount of prescribed continuous gastric carbohydrate administration from start of lead-in trial (g/day)	Baseline through treatment completion, up to 3 years
Prescribed duration of continuous gastric carbohydrate administration	Change in prescribed duration of infusion of continuous gastric carbohydrate administration from start of lead-in trial (h/day)	Baseline through treatment completion, up to 3 years
Prescribed duration of nightly continuous gastric carbohydrate administration	Change in prescribed duration of infusion of nightly (8 pm - 8 am) continuous gastric carbohydrate administration from start of lead-in trial (h/day)	Baseline through treatment completion, up to 3 years

Collaborators and Investigators

• Sponsor: Zealand Pharma
• Investigators: Study Director: Jelena Ivkovic, MD, Zealand Pharma

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2019
• Primary Completion (Estimated): January 1, 2025
• Study Completion (Estimated): January 1, 2025

Study Registration Dates

• First Submitted: May 2, 2019
• First Submitted That Met QC Criteria: May 4, 2019
• First Posted (Actual): May 7, 2019

Study Record Updates

• Last Update Posted (Actual): April 8, 2024
• Last Update Submitted That Met QC Criteria: April 5, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Glucose Metabolism Disorders; Metabolic Diseases; Infant, Newborn, Diseases; Pancreatic Diseases; Hypoglycemia; Hyperinsulinism; Congenital Hyperinsulinism; Nesidioblastosis
• Other Study ID Numbers: ZP4207-17106

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No