Clinical trial NCT03777176: A Two-Period Open-label Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism
Official title: A Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Congenital Hyperinsulinism
Lead sponsor: Zealand Pharma
Sponsor class: INDUSTRY
First posted: 2018-12-17
Enrollment: 32
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The objective of the trial is to evaluate the efficacy and safety of dasiglucagon administered as a subcutaneous (SC) infusion in reducing hypoglycemia in children with CHI.


Eligibility criteria: Inclusion Criteria:

* Established and documented diagnosis of CHI based on standard of care
* Experiencing ≥3 events of hypoglycemia per week (plasma glucose \[PG\] \<70 mg/dL \[\<3.9 mmol/L\]) according to the investigator's evaluation
* Previously undergone near-total pancreatectomy or being treated with a non-surgical approach, having been evaluated as not eligible for pancreatic surgery
* If somatostatin analogues or sirolimus are used, the therapy should be well established as judged by the investigator, especially when considering their biological half-life

Exclusion Criteria:

* Previous administration of dasiglucagon
* Known or suspected allergy to the trial drug or related products
* Previous participation (randomization) in this trial
* Circulatory instability requiring supportive medication or presence of pheochromocytoma
* Requires exogenous insulin
* Body weight of \<4 kg (8.8 lbs.)
* Documented HbA1c ≥7% subsequent to near-total pancreatectomy and within 6 months prior to screening
* Known or suspected presence of significant central nervous system disease/injury such that in the investigator's opinion will affect trial participation
* Use of systemic corticosteroids, e.g., hydrocortisone \>20 mg/m2 body surface area or equivalent in the 5 days before screening
* Use of anti-inflammatory biological agents, or other immune modulating agents in the 3 months prior to screening
* Any clinically significant abnormality identified on echocardiogram that in the opinion of the investigator would affect the patient's ability to participate in the trial
* Any recognized clotting or bleeding disorders
* Has participated in an interventional clinical trial (investigational or marketed product) within 3 months of screening or 5 half-lives of the drug under investigation (whichever comes first), or plans to participate in another clinical trial.

Primary outcomes:
measure: Hypoglycemia Episode Rate
timeFrame: Baseline, Week 2-4 (Treatment Period 1)
Secondary outcomes:
measure: Increase in Fasting Tolerance
timeFrame: Baseline, Week 2-4 (Treatment Period 1)
measure: Percent Time in Range 70-180 mg/dL
timeFrame: Baseline, Week 2-4 (Treatment Period 1)
measure: Clinically Significant Hypoglycemia Episode Rates
timeFrame: Baseline, Week 2-4 (Treatment Period 1)
measure: Total Amount of Gastric Carbohydrates Administered to Treat Hypoglycemia
timeFrame: Baseline, Week 2-4 (Treatment Period 1)
measure: Rate of Gastric Carbohydrates Administrations to Treat Hypoglycemia
timeFrame: Baseline, Week 2-4 (Treatment Period 1)
measure: Extent of Hypoglycemia
timeFrame: Baseline, Week 2-4 (Treatment Period 1)
measure: Amount of Nightly Gastric Carbohydrates Administered
timeFrame: Baseline, Week 2-4 (Treatment Period 1)
measure: Total Amount of Gastric Carbohydrates Administered
timeFrame: Baseline, Week 2-4 (Treatment Period 1)
measure: Percent Time in Hypoglycemia
timeFrame: Baseline, Week 2-4 (Treatment Period 1)
measure: Rate of Hypoglycemic Episodes
timeFrame: Baseline, Week 2-4 (Treatment Period 1)
measure: Percent Time in Hypoglycemia in Treatment Period 2
timeFrame: Baseline, Week 6-8 (Treatment Period 2)
measure: Rate of Gastric Carbohydrates Administrations to Treat Hypoglycemia in Treatment Period 2
timeFrame: Baseline, Week 6-8 (Treatment Period 2)
measure: Rate of Hypoglycemic Episodes in Treatment Period 2
timeFrame: Baseline, Week 6-8 (Treatment Period 2)
measure: Rate of Clinically Significant Hypoglycemia Episodes in Treatment Period 2
timeFrame: Baseline, Week 6-8 (Treatment Period 2)
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'sHospital 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: Charlotte Teglman Schioeler
role: STUDY_DIRECTOR
References: