Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon in Children With Congenital Hyperinsulinism
Study Details
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Dasiglucagon open-label Dasiglucagon treatment as sc infusion starting at 10 µg/hr on top of standard of care |
Drug: dasiglucagon
Glucagon analog
Other Names:
|
Outcome Measures
Primary Outcome Measures
- 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
- 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)
Eligibility Criteria
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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Children's Hospital of Colorado | Aurora | Colorado | United States | 13123 |
| 2 | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | United States | 19104 |
| 3 | Cook Children's Medical Center | Fort Worth | Texas | United States | 76104 |
| 4 | University Hospital Düsseldorf, Department of Pediatrics | Düsseldorf | Germany | 40225 | |
| 5 | Otto von Guericke University Magdeburg, Department of Pediatrics | Magdeburg | Germany | 39120 | |
| 6 | Hadassah Medical Center | Jerusalem | Israel | 9765422 | |
| 7 | NHS Greater Glasgow and Clyde | Glasgow | United Kingdom | ||
| 8 | Alder Hey Children's Hospital NHS Foundation Trust | Liverpool | United Kingdom | ||
| 9 | Great Osmond Street Hospital for Children NHS Foundation Trust | London | United Kingdom | ||
| 10 | Central Manchester University Hospital NHS Foundation Trust | Manchester | United Kingdom |
Sponsors and Collaborators
- Zealand Pharma
Investigators
- Study Director: Have Andersen Aliu, MSc, PhD, Zealand Pharma
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- ZP4207-17106