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

NCT03777176 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: ZP4207-17109
• Study Type: interventional
• Target: 32
• Locations: 4 countries
• Sites: 10

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.

Conditions

Congenital Hyperinsulinism

Outcome Measures

Primary Outcomes (1)

• Hypoglycemia Episode Rate

  Hypoglycemia episode rate, defined as average weekly number of hypoglycemic episodes (PG \<70 mg/dL \[3.9 mmol/L\]) during Weeks 2-4 as detected by self-monitored plasma glucose (SMPG). The Week 2-4 value is the average weekly number of hypoglycemic episodes across the last 3 weeks of the treatment period. The change from Baseline to Week 2-4 is also provided. The Baseline value is the average weekly number of hypoglycemic episodes during the 2-week baseline period.

  Baseline, Week 2-4 (Treatment Period 1)

Secondary Outcomes (14)

• Increase in Fasting Tolerance

  Baseline, Week 2-4 (Treatment Period 1)

• Percent Time in Range 70-180 mg/dL

  Baseline, Week 2-4 (Treatment Period 1)

• Clinically Significant Hypoglycemia Episode Rates

  Baseline, Week 2-4 (Treatment Period 1)

• Total Amount of Gastric Carbohydrates Administered to Treat Hypoglycemia

  Baseline, Week 2-4 (Treatment Period 1)

• Rate of Gastric Carbohydrates Administrations to Treat Hypoglycemia

  Baseline, Week 2-4 (Treatment Period 1)

Study Arms (2)

Standard of Care + dasiglucagon

EXPERIMENTAL

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

Drug: Dasiglucagon; Other: Standard of Care

Standard of Care Only

OTHER

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

Drug: Dasiglucagon; Other: Standard of Care

Interventions

Dasiglucagon DRUG

Glucagon analog

Also known as: ZP4207

Standard of Care + dasiglucagon; Standard of Care Only

Standard of Care OTHER

Standard of care according to site and/or country

Standard of Care + dasiglucagon; Standard of Care Only

Eligibility Criteria

• Age: 3 Months - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Zealand Pharma: lead

Study Sites (10)

Children's Hospital of Colorado

Aurora, Colorado, 13123, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

University Hospital Düsseldorf, Department of Pediatrics

Düsseldorf, 40225, Germany

Location

Otto von Guericke University Magdeburg, Department of Pediatrics

Magdeburg, 39120, Germany

Location

Hadassah Medical Center

Jerusalem, 9765422, Israel

Location

NHS Greater Glasgow and Clyde

Glasgow, United Kingdom

Location

Alder Hey Children'sHospital NHS Foundation Trust

Liverpool, United Kingdom

Location

Great Osmond Street Hospital for Children NHS Foundation Trust

London, United Kingdom

Location

Central Manchester University Hospital NHS Foundation Trust

Manchester, United Kingdom

Location

MeSH Terms

Conditions

Congenital Hyperinsulinism

Interventions

dasiglucagon; Standard of Care

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperinsulinism; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Hypoglycemia

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Results Point of Contact

• Title: Sune Birch
• Organization: Zealand Pharma

SBirch@zealandpharma.com

+45 5060 3864

Study Officials

• Charlotte Teglman Schioeler

  Zealand Pharma

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 13, 2018
• First Posted: December 17, 2018
• Study Start: February 7, 2019
• Primary Completion: August 31, 2020
• Study Completion: October 5, 2020
• Last Updated: December 13, 2023
• Results First Posted: December 13, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

IL (1); US (3); DE (2); GB (4)