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

Phase 3

Active, not recruiting

• Conditions: Congenital Hyperinsulinism
• Interventions: Drug: dasiglucagon

• Registration Number: NCT03941236
• Lead Sponsor: Zealand Pharma

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-05-01
• Study First Submitted: 2019-05-02
• Study First Submitted QC: 2019-05-04
• Study First Posted: 2019-05-07
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-05
• Last Update Posted: 2024-04-08
• Primary Completion: 2025-01-01
• Study Completion: 2025-01-01

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 44

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)

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Dasiglucagon open-label	dasiglucagon	Dasiglucagon treatment as sc infusion starting at 10 µg/hr on top of standard of care

Primary Outcome Measures

Name	Time	Method
Adverse Events	Baseline through treatment completion, up to 3 years	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

Secondary Outcome Measures

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

Trial Locations

Locations (10)

University Hospital Düsseldorf, Department of Pediatrics; 🇩🇪 Düsseldorf, Germany

Cook Children's Medical Center; 🇺🇸 Fort Worth, Texas, United States

Hadassah Medical Center; 🇮🇱 Jerusalem, Israel

NHS Greater Glasgow and Clyde; 🇬🇧 Glasgow, United Kingdom

Otto von Guericke University Magdeburg, Department of Pediatrics; 🇩🇪 Magdeburg, Germany

Alder Hey Children's Hospital NHS Foundation Trust; 🇬🇧 Liverpool, United Kingdom

Central Manchester University Hospital NHS Foundation Trust; 🇬🇧 Manchester, United Kingdom

Great Osmond Street Hospital for Children NHS Foundation Trust; 🇬🇧 London, United Kingdom

Children's Hospital of Colorado; 🇺🇸 Aurora, Colorado, United States

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States