Congenital HyperinsulinismPhase 2/3NCT04172441
Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism
A Phase 2/3 study testing dasiglucagon in people with Congenital Hyperinsulinism, run by Zealand Pharma at 6 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 7 to 364 days
- Conditions the study is for
- Congenital Hyperinsulinism
- What is being tested?
- dasiglucagon
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 6 sitesin 4 countries
- Study dates
- 19 Jun 2020to 7 Mar 2022
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 364 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
6 sites in 4 countries.
6 sites in 4 countries
United States2 sites
Cook Children's Medical Center
Fort Worth, Texas, 76104
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Germany2 sites
University Children's Hospital
Düsseldorf, 40225
University Hospital, Magdeburg
Magdeburg, 39120
United Kingdom1 site
Manchester University NHS Foundation Trust
Manchester, M13 9WL
About this study
Official title: A Randomized Trial in 2 Parts: Double-Blind, Placebo-Controlled, Crossover Part 1 and Open-label Part 2, Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism
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
- Being tested
- dasiglucagon, Placebo
- Medical subject headings
- Congenital Hyperinsulinism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
dasiglucagonDrug
Also called ZP4207
Glucagon analogue
PlaceboDrug
Placebo for dasiglucagon
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: 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). Receives: Drug: dasiglucagon; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Hours 36-48 after initiation of trial drug (Part 1)
Secondary outcomes (21)
Carbohydrates Administered
Total amount (g) of carbohydrates administered during the crossover part of the trial (dasiglucagon or placebo administration) per day.
Measured: 0 to 48 hours after initiation of trial drug
Mean Intravenous Glucose Infusion Rate
Mean IV GIR for each 48-hour treatment period during Part 1, the crossover part of the trial (dasiglucagon or placebo administration).
Measured: 48 hours after initiation of trial drug (Part 1)
Mean Intravenous Glucose Infusion Rate Below 10 mg/kg/Minute
Mean IV GIR below 10 mg/kg/minute in the last 12 hours of each treatment period during Part 1, the crossover part of the trial (dasiglucagon or placebo administration) (yes/no)
Measured: Hours 36-48 after initiation of trial drug (Part 1)
Time to Complete Weaning Off Intravenous Glucose
Time in days to complete weaning off IV glucose administration during Part 2, defined as the first point in time when the patient had been off IV glucose administration for at least 12 hours.
Measured: Days 5 to 25 (Part 2)
Hypoglycemia Event Rate in Part 2
Hypoglycemia event rate, defined as number of hypoglycemic events when PG was <70 mg/dL (or 3.9 mmol/L), as detected by self-monitored plasma glucose.
Measured: Days 5 to 25
Clinically Significant Hypoglycemia Events in Part 2
Clinically significant hypoglycemia event rate, defined as number of events when PG was <54 mg/dL (3.0 mmol/L), as detected by self-monitored plasma glucose.
Measured: Days 5 to 25
Time to Actual Hospital Discharge
Time in days to actual hospital discharge defined as the time from first exposure to the study drug in Part 2 to discharge from hospital.
Measured: Days 5 to 25
Time to Pancreatic Surgery
Time (days) to pancreatic surgery (sub-total or total pancreatectomy with a cutoff of ≥95%).
Measured: Days 5 to 25
Carbohydrates Administered
Total amount (g) of carbohydrates administered (regardless of the route) per day.
Measured: Days 5 to 25
Carbohydrates Administered Intravenously
Amount (g) of carbohydrates administered via IV glucose infusion or bolus or total parenteral nutrition. This secondary endpoint was intended to account only for carbohydrates administered via IV glucose infusion or bolus. It was not possible to differentiate between carbohydrates administered via IV glucose infusion or bolus and carbohydrates administered as being, or not being, part of total parenteral nutrition from the collected data. This endpoint was expanded to include both.
Measured: Days 5 to 25
Carbohydrates Administered Parenterally
Amount (g) of carbohydrates administered as part of total parenteral nutrition.
Measured: Days 5 to 25
Carbohydrates Administered Orally
Amount (g) of carbohydrates administered via oral route.
Measured: Days 5 to 25
Carbohydrates Administered Via Gastric Feed
Amount (g) of carbohydrates administered via nasogastric tube or gastrostomy.
Measured: Days 5 to 25
Time in Range in Part 2
Percent time in range (PG between 70 to 180 mg/dL [3.9-10.0 mmol/L]) as measured by continuous glucose monitoring.
Measured: Days 5 to 25
Time in Hypoglycemia in Part 2
Percent time in hypoglycemia (when PG was <70 mg/dL [or 3.9 mmol/L]) as measured by continuous glucose monitoring.
Measured: Days 5 to 25
Time in Clinically Significant Hypoglycemia in Part 2
Percent time in clinically significant hypoglycemia (when PG was <54 mg/dL [or 3.0 mmol/L]) as measured by continuous glucose monitoring.
Measured: Days 5 to 25
Hypoglycemia Episodes in Part 2
Rate of hypoglycemia episodes, defined as number of episodes per week when PG was <70 mg/dL (3.9 mmol/L) for 15 minutes or more, as measured by continuous glucose monitoring.
Measured: Days 5 to 25
Clinically Significant Hypoglycemia Episodes in Part 2
Rate of clinically significant hypoglycemia episodes, defined as number of episodes per week when was <54 mg/dL (3.0 mmol/L) for 15 minutes or more, as measured by continuous glucose monitoring.
Measured: Days 5 to 25
Extent of Hypoglycemia in Part 2
Extent of hypoglycemia (defined as the area over the glucose curve [AOCglucose] below 70 mg/dL [3.9 mmol/L]) as measured by continuous glucose monitoring.
Measured: Days 5 to 25
Extent of Clinically Significant Hypoglycemia in Part 2
Extent of clinically significant hypoglycemia (defined as the area over the glucose curve [AOCglucose] below 54 mg/dL [3.0 mmol/L]) as measured by continuous glucose monitoring, divided by the total duration in hours of continuous glucose monitoring.
Measured: Days 5 to 25
Time in Hyperglycemia in Part 2
Percent time in hyperglycemia (when PG was >180 mg/dL [10.0 mmol/L]), as measured by continuous glucose monitoring.
Measured: Days 5 to 25
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2019
- Study start19 Jun 2020
- Primary completion17 Feb 2022
- Study completion7 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Congenital HyperinsulinismStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zealand Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jelena Ivkovic, MD, Study director, Zealand Pharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04172441
- Sponsor's protocol ID
- ZP4207-17103
- Other IDs
- 2017-004545-24, 2017
- Status last verified
- Mar 2025
- First submitted
- 18 Nov 2019
- Last updated
- 14 Mar 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Trial in 2 Parts: Double-Blind, Placebo-Controlled, Crossover Part 1 and Open-label Part 2, Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism
- ClinicalTrials.gov ID
- NCT04172441
- Acronym / sponsor ID
- ZP4207-17103
- Conditions
- Congenital Hyperinsulinism
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Zealand PharmaIndustry (pharma, biotech, devices)
- Study start
- 19 Jun 2020 (actual)
- Primary completion
- 17 Feb 2022 (actual)
- Study completion
- 7 Mar 2022 (actual)
- First posted
- 21 Nov 2019 (actual)
- Results
- Posted 14 Mar 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| dasiglucagon first then placebo | Experimental | Drug: dasiglucagon, Drug: Placebo48 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). |
| placebo first then dasiglucagon | Experimental | Drug: dasiglucagon, Drug: Placebo48 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). |
Who may qualify
- Ages
- 7 to 364 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- CHI diagnosis established based on the following:
- 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
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| dasiglucagonAlso: ZP4207Glucagon analogue | Drug | dasiglucagon first then placebo, placebo first then dasiglucagon |
| PlaceboPlacebo for dasiglucagon | Drug | dasiglucagon first then placebo, placebo first then dasiglucagon |
Primary outcome
Mean Intravenous Glucose Infusion Rate
Time frame: Hours 36-48 after initiation of trial drug (Part 1)
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).
21 secondary outcomes
Carbohydrates Administered
Time frame: 0 to 48 hours after initiation of trial drug
Mean Intravenous Glucose Infusion Rate
Time frame: 48 hours after initiation of trial drug (Part 1)
Mean Intravenous Glucose Infusion Rate Below 10 mg/kg/Minute
Time frame: Hours 36-48 after initiation of trial drug (Part 1)
Time to Complete Weaning Off Intravenous Glucose
Time frame: Days 5 to 25 (Part 2)
Hypoglycemia Event Rate in Part 2
Time frame: Days 5 to 25
Clinically Significant Hypoglycemia Events in Part 2
Time frame: Days 5 to 25
Time to Actual Hospital Discharge
Time frame: Days 5 to 25
Time to Pancreatic Surgery
Time frame: Days 5 to 25
Carbohydrates Administered
Time frame: Days 5 to 25
Carbohydrates Administered Intravenously
Time frame: Days 5 to 25
Carbohydrates Administered Parenterally
Time frame: Days 5 to 25
Carbohydrates Administered Orally
Time frame: Days 5 to 25
Carbohydrates Administered Via Gastric Feed
Time frame: Days 5 to 25
Time in Range in Part 2
Time frame: Days 5 to 25
Time in Hypoglycemia in Part 2
Time frame: Days 5 to 25
Time in Clinically Significant Hypoglycemia in Part 2
Time frame: Days 5 to 25
Hypoglycemia Episodes in Part 2
Time frame: Days 5 to 25
Clinically Significant Hypoglycemia Episodes in Part 2
Time frame: Days 5 to 25
Extent of Hypoglycemia in Part 2
Time frame: Days 5 to 25
Extent of Clinically Significant Hypoglycemia in Part 2
Time frame: Days 5 to 25
Time in Hyperglycemia in Part 2
Time frame: Days 5 to 25
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Mar 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jelena Ivkovic, MD study director
Zealand Pharma
6 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing dasiglucagon in people with Congenital Hyperinsulinism, run by Zealand Pharma at 6 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Germany, Israel, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Mar 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean Intravenous Glucose Infusion Rate (time frame: Hours 36-48 after initiation of trial drug (Part 1)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Mar 2025.