Congenital Hyperinsulinism
Conditions
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 a subcutaneous (SC) infusion in children with CHI.
Interventions
DRUGdasiglucagon
Glucagon analog
Sponsors
Zealand Pharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Weeks to 13 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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) |
| Time in hypoglycemia | Baseline through treatment completion, up to 3 years | Continuous glucose monitoring (CGM) percent time \<70 mg/dL (3.9 mmol/L) |
| 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 |
| 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) |
| 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) |
| 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 |
| 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) |
Countries
Germany, Israel, United Kingdom, United States
Contacts
STUDY_DIRECTORClinical Trial Information Desk
Zealand Pharma
Outcome results
None listed