Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism

NCT04172441 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: ZP4207-171032017-004545-24
• Study Type: interventional
• Target: 12
• Locations: 4 countries
• Sites: 6

Brief Summary

The objective of the trial is to evaluate the efficacy of dasiglucagon in reducing glucose requirements in children with persistent congenital hyperinsulinism (CHI) requiring continuous intravenous (IV) glucose administration to prevent/manage hypoglycemia.

Conditions

Congenital Hyperinsulinism

Outcome Measures

Primary Outcomes (1)

• Mean Intravenous Glucose Infusion Rate

  Mean intravenous (IV) glucose infusion rate (GIR) in the last 12 hours of each treatment period during Part 1, the crossover part of the trial (dasiglucagon or placebo administration).

  Hours 36-48 after initiation of trial drug (Part 1)

Secondary Outcomes (21)

• Carbohydrates Administered

  0 to 48 hours after initiation of trial drug

• Mean Intravenous Glucose Infusion Rate

  48 hours after initiation of trial drug (Part 1)

• Mean Intravenous Glucose Infusion Rate Below 10 mg/kg/Minute

  Hours 36-48 after initiation of trial drug (Part 1)

• Time to Complete Weaning Off Intravenous Glucose

  Days 5 to 25 (Part 2)

• Hypoglycemia Event Rate in Part 2

  Days 5 to 25

Study Arms (2)

dasiglucagon first then placebo

EXPERIMENTAL

48 hours of dasiglucagon subcutaneous (sc) infusion starting at 10 µg/hour with crossover to 48 hours placebo sc infusion (part 1) followed by 21 days of dasiglucagon sc infusion (part 2).

Drug: dasiglucagon; Drug: Placebo

placebo first then dasiglucagon

EXPERIMENTAL

48 hours of placebo sc infusion with crossover to 48 hours dasiglucagon sc infusion starting at 10 µg/hour (part 1) followed by 21 days of dasiglucagon sc infusion (part 2).

Drug: dasiglucagon; Drug: Placebo

Interventions

dasiglucagon DRUG

Glucagon analogue

Also known as: ZP4207

dasiglucagon first then placebo; placebo first then dasiglucagon

Placebo DRUG

Placebo for dasiglucagon

dasiglucagon first then placebo; placebo first then dasiglucagon

Eligibility Criteria

• Age: 7 Days - 364 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• CHI diagnosis established based on the following:
• Hyperinsulinemia: plasma insulin above the limit of detection of the assay documented during an event of hypoglycemia, and/or
• Hypofattyacidemia: plasma free fatty acid \<1.7 mmol/L, and/or
• Hypoketonemia: Beta-hydroxybutyrate \<1.8 mmol/L, and/or
• Glycemic response: an increase in plasma glucose (PG) of \>30 mg/dL (1.7 mmol/L) after 1 mg IV or intramuscular (IM) glucagon administration
• Male or female, age ≥7 days and \<12 months at screening
• Body weight of ≥2.0 kg (4.4 lbs.)
• Continuous IV glucose requirement to prevent hypoglycemia

You may not qualify if:

• Is suspected of having a transient form of CHI (e.g., transient hyperinsulinism due to maternal diabetes or perinatal stress)
• Was born preterm below 34 weeks of gestational age
• Presence of hypertension or hypotension, including circulatory instability requiring supportive medication or presence of pheochromocytoma
• Known or suspected presence of severe brain damage
• Evidence of metabolic, endocrine, or syndromic causes of hypoglycemia not due to hyperinsulinism
• Use of systemic corticosteroids, e.g., hydrocortisone \>20 mg/m\^2 body surface area or equivalent within 5 days before screening
• Prior use of lanreotide, sirolimus (mechanistic target of rapamycin \[mTOR\] inhibitors), anti-inflammatory biological agents, or other immune modulating agents. Prior use of octreotide is allowed after a minimum of 48 hour washout before randomization.
• Any clinically significant abnormality identified on echocardiogram that in the opinion of the investigator would affect the subject's ability to participate in the trial
• Any recognized clotting or bleeding disorder
• The use of prescription or non-prescription medications known to cause QT prolongation

Sponsors & Collaborators

• Zealand Pharma: lead

Study Sites (6)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

University Children's Hospital

Düsseldorf, 40225, Germany

Location

University Hospital, Magdeburg

Magdeburg, 39120, Germany

Location

Hadassah Medical Center

Jerusalem, 9765422, Israel

Location

Manchester University NHS Foundation Trust

Manchester, M13 9WL, United Kingdom

Location

MeSH Terms

Conditions

Congenital Hyperinsulinism

Interventions

dasiglucagon

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

Results Point of Contact

• Title: Charlotte Teglman Schiøler
• Organization: Zealand Pharma A/S

cschioler@zealandpharma.com

+45 5060 3722

Study Officials

• Jelena Ivkovic, MD

  Zealand Pharma

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 18, 2019
• First Posted: November 21, 2019
• Study Start: June 19, 2020
• Primary Completion: February 17, 2022
• Study Completion: March 7, 2022
• Last Updated: March 14, 2025
• Results First Posted: March 14, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

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