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

Phase 3Active Not RecruitingNCT03941236

Zealand Pharma42 enrolled

Overview

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 a subcutaneous (SC) infusion in children with CHI.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Congenital Hyperinsulinism

Interventions

dasiglucagonDRUG

Glucagon analog

Eligibility

Sex: ALLMin age: 5 WeeksMax age: 13 Years

Medical Language ↔ Plain English

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.

Locations (10)

Children's Hospital Colorado

Aurora, Colorado, United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Cook Children's Endocrinology and Diabetes Clinic

Fort Worth, Texas, United States

Outcomes

Primary Outcomes

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

Time frame: Baseline through treatment completion, up to 3 years

Secondary Outcomes

Amount of gastric carbohydrates administered to treat hypoglycemia

Total amount of gastric carbohydrates administered via nasogastric tube or gastrostomy per week to treat hypoglycemia

Time frame: Baseline through treatment completion, up to 3 years

Nasogastric (NG) tube or gastrostomy removal

Time to removal of NG tube or gastrostomy

Time frame: Baseline through treatment completion, up to 3 years

Pancreatic surgery

Time to pancreatic surgery (sub-total or total pancreatectomy)

Time frame: Baseline through treatment completion, up to 3 years

Time in hypoglycemia

Continuous glucose monitoring (CGM) percent time \<70 mg/dL (3.9 mmol/L)

Time frame: 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

Time frame: 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

Data from ClinicalTrials.gov

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University Hospital Düsseldorf, Department of Pediatrics

Düsseldorf, Germany

Otto von Guericke University Magdeburg, Department of Pediatrics

Magdeburg, Germany

Hadassah Medical Center

Jerusalem, Israel

NHS Greater Glasgow and Clyde

Glasgow, United Kingdom

Alder Hey Children's Hospital NHS Foundation Trust

Liverpool, United Kingdom

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom

Manchester University NHS Foundation Trust

Manchester, United Kingdom

Time frame: Baseline through treatment completion, up to 3 years

Gastric carbohydrate administrations

Number of gastric carbohydrate administrations (nasogastric tube or gastrostomy) to treat hypoglycemia

Time frame: 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

Time frame: 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)

Time frame: 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)

Time frame: 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

Time frame: Baseline through treatment completion, up to 3 years

Somatostatin analog dose

Reduction in somatostatin analog dose from start of lead-in trial

Time frame: 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)

Time frame: 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)

Time frame: 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)

Time frame: Baseline through treatment completion, up to 3 years