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

Primary Purpose

Congenital Hyperinsulinism

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
dasiglucagon
Sponsored by
Zealand Pharma
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Hyperinsulinism

Eligibility Criteria

6 Weeks - 13 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's Hospital of Colorado
  • Children's Hospital of Philadelphia
  • Cook Children's Medical Center
  • University Hospital Düsseldorf, Department of Pediatrics
  • Otto von Guericke University Magdeburg, Department of Pediatrics
  • Hadassah Medical Center
  • NHS Greater Glasgow and Clyde
  • Alder Hey Children's Hospital NHS Foundation Trust
  • Great Osmond Street Hospital for Children NHS Foundation Trust
  • Central Manchester University Hospital NHS Foundation Trust

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Dasiglucagon open-label

Arm Description

Dasiglucagon treatment as sc infusion starting at 10 µg/hr on top of standard of care

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Amount of gastric carbohydrates administered to treat hypoglycemia
Total amount of gastric carbohydrates administered via nasogastric tube or gastrostomy per week to treat hypoglycemia
Nasogastric (NG) tube or gastrostomy removal
Time to removal of NG tube or gastrostomy
Pancreatic surgery
Time to pancreatic surgery (sub-total or total pancreatectomy)
Time in hypoglycemia
Continuous glucose monitoring (CGM) percent time <70 mg/dL (3.9 mmol/L)
Hypoglycemia episodes
Rate of CGM-detected hypoglycemia episodes <70 mg/dL (3.9 mmol/L) for 15 minutes or more
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
Gastric carbohydrate administrations
Number of gastric carbohydrate administrations (nasogastric tube or gastrostomy) to treat hypoglycemia
Nightly gastric carbohydrate administrations
Number of nightly (midnight to 6 am) gastric carbohydrate administrations (nasogastric tube or gastrostomy) to treat hypoglycemia
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)
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)
Diazoxide dose
Reduction in diazoxide dose in mg/kg body weight/day from start of lead-in trial
Somatostatin analog dose
Reduction in somatostatin analog dose from start of lead-in trial
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)
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)
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)

Full Information

First Posted
May 2, 2019
Last Updated
September 26, 2023
Sponsor
Zealand Pharma
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1. Study Identification

Unique Protocol Identification Number
NCT03941236
Brief Title
Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon in Children With Congenital Hyperinsulinism
Official Title
An Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
May 1, 2019 (Actual)
Primary Completion Date
April 2024 (Anticipated)
Study Completion Date
April 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Zealand Pharma

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Hyperinsulinism

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
44 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Dasiglucagon open-label
Arm Type
Experimental
Arm Description
Dasiglucagon treatment as sc infusion starting at 10 µg/hr on top of standard of care
Intervention Type
Drug
Intervention Name(s)
dasiglucagon
Other Intervention Name(s)
ZP4207
Intervention Description
Glucagon analog
Primary Outcome Measure Information:
Title
Adverse Events
Description
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 Outcome Measure Information:
Title
Amount of gastric carbohydrates administered to treat hypoglycemia
Description
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
Title
Nasogastric (NG) tube or gastrostomy removal
Description
Time to removal of NG tube or gastrostomy
Time Frame
Baseline through treatment completion, up to 3 years
Title
Pancreatic surgery
Description
Time to pancreatic surgery (sub-total or total pancreatectomy)
Time Frame
Baseline through treatment completion, up to 3 years
Title
Time in hypoglycemia
Description
Continuous glucose monitoring (CGM) percent time <70 mg/dL (3.9 mmol/L)
Time Frame
Baseline through treatment completion, up to 3 years
Title
Hypoglycemia episodes
Description
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
Title
Clinically significant episodes of hypoglycemia
Description
Rate of clinically significant CGM-detected hypoglycemia episodes <54 mg/dL (3.0 mmol/L) for 15 minutes or more
Time Frame
Baseline through treatment completion, up to 3 years
Title
Gastric carbohydrate administrations
Description
Number of gastric carbohydrate administrations (nasogastric tube or gastrostomy) to treat hypoglycemia
Time Frame
Baseline through treatment completion, up to 3 years
Title
Nightly gastric carbohydrate administrations
Description
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
Title
Extent of hypoglycemia
Description
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
Title
Extent of clinically significant hypoglycemia
Description
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
Title
Diazoxide dose
Description
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
Title
Somatostatin analog dose
Description
Reduction in somatostatin analog dose from start of lead-in trial
Time Frame
Baseline through treatment completion, up to 3 years
Title
Prescribed amount of continuous gastric carbohydrate administration
Description
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
Title
Prescribed duration of continuous gastric carbohydrate administration
Description
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
Title
Prescribed duration of nightly continuous gastric carbohydrate administration
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Weeks
Maximum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jelena Ivkovic, MD
Organizational Affiliation
Zealand Pharma
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
13123
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Cook Children's Medical Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
University Hospital Düsseldorf, Department of Pediatrics
City
Düsseldorf
ZIP/Postal Code
40225
Country
Germany
Facility Name
Otto von Guericke University Magdeburg, Department of Pediatrics
City
Magdeburg
ZIP/Postal Code
39120
Country
Germany
Facility Name
Hadassah Medical Center
City
Jerusalem
ZIP/Postal Code
9765422
Country
Israel
Facility Name
NHS Greater Glasgow and Clyde
City
Glasgow
Country
United Kingdom
Facility Name
Alder Hey Children's Hospital NHS Foundation Trust
City
Liverpool
Country
United Kingdom
Facility Name
Great Osmond Street Hospital for Children NHS Foundation Trust
City
London
Country
United Kingdom
Facility Name
Central Manchester University Hospital NHS Foundation Trust
City
Manchester
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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