A Two-Period Open-label Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism

NCT ID: NCT03777176

Last Updated: 2023-12-13

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

32 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-02-07

Study Completion Date

2020-10-05

Brief Summary

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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.

Detailed Description

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Conditions

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Congenital Hyperinsulinism

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Standard of Care + dasiglucagon

4 weeks (Treatment Period 1) + 4 weeks (Treatment Period 2) of dasiglucagon treatment as an SC infusion starting at 10 µg/hr on top of standard of care

Group Type EXPERIMENTAL

Dasiglucagon

Intervention Type DRUG

Glucagon analog

Standard of Care

Intervention Type OTHER

Standard of care according to site and/or country

Standard of Care Only

4 weeks (Treatment Period 1) of standard of care only + 4 weeks (Treatment Period 2) of dasiglucagon treatment as an SC infusion starting at 10 µg/hr on top of standard of care

Group Type OTHER

Dasiglucagon

Intervention Type DRUG

Glucagon analog

Standard of Care

Intervention Type OTHER

Standard of care according to site and/or country

Interventions

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Dasiglucagon

Glucagon analog

Intervention Type DRUG

Standard of Care

Standard of care according to site and/or country

Intervention Type OTHER

Other Intervention Names

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ZP4207

Eligibility Criteria

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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.
Minimum Eligible Age

3 Months

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Zealand Pharma

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Charlotte Teglman Schioeler

Role: STUDY_DIRECTOR

Zealand Pharma

Locations

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Children's Hospital of Colorado

Aurora, Colorado, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Cook Children's Medical Center

Fort Worth, Texas, United States

Site Status

University Hospital Düsseldorf, Department of Pediatrics

Düsseldorf, , Germany

Site Status

Otto von Guericke University Magdeburg, Department of Pediatrics

Magdeburg, , Germany

Site Status

Hadassah Medical Center

Jerusalem, , Israel

Site Status

NHS Greater Glasgow and Clyde

Glasgow, , United Kingdom

Site Status

Alder Hey Children'sHospital NHS Foundation Trust

Liverpool, , United Kingdom

Site Status

Great Osmond Street Hospital for Children NHS Foundation Trust

London, , United Kingdom

Site Status

Central Manchester University Hospital NHS Foundation Trust

Manchester, , United Kingdom

Site Status

Countries

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United States Germany Israel United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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ZP4207-17109

Identifier Type: -

Identifier Source: org_study_id