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

Congenital Hyperinsulinism Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03941236
• Other study ID #: ZP4207-17106
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: May 1, 2019
• Est. completion date: January 1, 2025

Study information

• Verified date: April 2024
• Source: Zealand Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 44
• Est. completion date: January 1, 2025
• Est. primary completion date: January 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Weeks to 13 Years

Eligibility

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.

Related Conditions & MeSH terms

• Congenital Hyperinsulinism
• Hyperinsulinism
• Nesidioblastosis

Intervention

Drug:
• dasiglucagon
Glucagon analog

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Zealand Pharma

Countries where clinical trial is conducted

United States,  Germany,  Israel,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	Nasogastric (NG) tube or gastrostomy removal	Time to removal of NG tube or gastrostomy	Baseline through treatment completion, up to 3 years
Secondary	Pancreatic surgery	Time to pancreatic surgery (sub-total or total pancreatectomy)	Baseline through treatment completion, up to 3 years
Secondary	Time in hypoglycemia	Continuous glucose monitoring (CGM) percent time <70 mg/dL (3.9 mmol/L)	Baseline through treatment completion, up to 3 years
Secondary	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
Secondary	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
Secondary	Gastric carbohydrate administrations	Number of gastric carbohydrate administrations (nasogastric tube or gastrostomy) to treat hypoglycemia	Baseline through treatment completion, up to 3 years
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Somatostatin analog dose	Reduction in somatostatin analog dose from start of lead-in trial	Baseline through treatment completion, up to 3 years
Secondary	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
Secondary	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
Secondary	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