Clinical trial NCT03941236: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Congenital Hyperinsulinism
Lead sponsor: Zealand Pharma
Sponsor class: INDUSTRY
First posted: 2019-05-07
Enrollment: 44
Sex: ALL
Minimum age (years): 6
Maximum age (years): 13
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Adverse Events
timeFrame: Baseline through treatment completion, up to 3 years
Secondary outcomes:
measure: Amount of gastric carbohydrates administered to treat hypoglycemia
timeFrame: Baseline through treatment completion, up to 3 years
measure: Nasogastric (NG) tube or gastrostomy removal
timeFrame: Baseline through treatment completion, up to 3 years
measure: Pancreatic surgery
timeFrame: Baseline through treatment completion, up to 3 years
measure: Time in hypoglycemia
timeFrame: Baseline through treatment completion, up to 3 years
measure: Hypoglycemia episodes
timeFrame: Baseline through treatment completion, up to 3 years
measure: Clinically significant episodes of hypoglycemia
timeFrame: Baseline through treatment completion, up to 3 years
measure: Gastric carbohydrate administrations
timeFrame: Baseline through treatment completion, up to 3 years
measure: Nightly gastric carbohydrate administrations
timeFrame: Baseline through treatment completion, up to 3 years
measure: Extent of hypoglycemia
timeFrame: Baseline through treatment completion, up to 3 years
measure: Extent of clinically significant hypoglycemia
timeFrame: Baseline through treatment completion, up to 3 years
measure: Diazoxide dose
timeFrame: Baseline through treatment completion, up to 3 years
measure: Somatostatin analog dose
timeFrame: Baseline through treatment completion, up to 3 years
measure: Prescribed amount of continuous gastric carbohydrate administration
timeFrame: Baseline through treatment completion, up to 3 years
measure: Prescribed duration of continuous gastric carbohydrate administration
timeFrame: Baseline through treatment completion, up to 3 years
measure: Prescribed duration of nightly continuous gastric carbohydrate administration
timeFrame: Baseline through treatment completion, up to 3 years
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Children's Hospital of Colorado
state: Colorado
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Düsseldorf
country: Germany
facility: University Hospital Düsseldorf, Department of Pediatrics
city: Magdeburg
country: Germany
facility: Otto von Guericke University Magdeburg, Department of Pediatrics
city: Jerusalem
country: Israel
facility: Hadassah Medical Center
city: Glasgow
country: United Kingdom
facility: NHS Greater Glasgow and Clyde
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's Hospital NHS Foundation Trust
city: London
country: United Kingdom
facility: Great Osmond Street Hospital for Children NHS Foundation Trust
city: Manchester
country: United Kingdom
facility: Central Manchester University Hospital NHS Foundation Trust
Study officials:
affiliation: Zealand Pharma
name: Jelena Ivkovic, MD
role: STUDY_DIRECTOR
References: