Congenital HyperinsulinismPhase 3NCT03777176
A Two-Period Open-label Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism
A Phase 3 study testing Dasiglucagon in people with Congenital Hyperinsulinism, run by Zealand Pharma at 10 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 months to 12 years
- Conditions the study is for
- Congenital Hyperinsulinism
- What is being tested?
- Dasiglucagon
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 10 sitesin 4 countries
- Study dates
- 7 Feb 2019to 5 Oct 2020
- Phase
- Phase 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 3 months to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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.)
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- 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.
Trial locations and contacts
10 sites in 4 countries.
10 sites in 4 countries
United Kingdom4 sites
NHS Greater Glasgow and Clyde
Glasgow
Alder Hey Children'sHospital NHS Foundation Trust
Liverpool
Great Osmond Street Hospital for Children NHS Foundation Trust
London
Central Manchester University Hospital NHS Foundation Trust
Manchester
United States3 sites
Children's Hospital of Colorado
Aurora, Colorado, 13123
Cook Children's Medical Center
Fort Worth, Texas, 76104
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Germany2 sites
University Hospital Düsseldorf, Department of Pediatrics
Düsseldorf, 40225
Otto von Guericke University Magdeburg, Department of Pediatrics
Magdeburg, 39120
About this study
Official title: A Two-Period, Open-label Trial 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 and safety of dasiglucagon administered as a subcutaneous (SC) infusion in reducing hypoglycemia in children with CHI.
- Conditions
- Congenital Hyperinsulinism
- Being tested
- Dasiglucagon, Standard of Care
- 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 analog
Standard of CareOther
Standard of care according to site and/or country
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: Drug: Dasiglucagon; Other: Standard of Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, Week 2-4 (Treatment Period 1)
Secondary outcomes (14)
Increase in Fasting Tolerance
Increase in fasting tolerance (time from beginning of meal to the beginning of the first continuous 15-minute continuous glucose monitoring reading <70 mg/dL [3.9 mmol/L]), or the time the test ended if a continuous 15-minute CGM reading <70 mg/dL was not reached. The change from Baseline to the End of Treatment Period 1 (Week 4) is also provided. The Baseline value is the value recorded at the Screening visit.
Measured: Baseline, Week 2-4 (Treatment Period 1)
Percent Time in Range 70-180 mg/dL
Percent time in range 70-180 mg/dL (3.9-10.0 mmol/L) during Weeks 2-4 as measured by continuous glucose monitoring. The Week 2-4 value is the average weekly percent time in range 70-180 mg/dL across the last 3 weeks of the treatment period. The change from Baseline to Week 2-4 is also provided. The Baseline value was calculated as the average percent time in range during the 14 days before randomization.
Measured: Baseline, Week 2-4 (Treatment Period 1)
Clinically Significant Hypoglycemia Episode Rates
Clinically significant hypoglycemia episode rates during Weeks 2-4, defined as average weekly number of clinically significant hypoglycemic episodes (PG <54 mg/dL [3.0 mmol/L]), as detected by SMPG. The Week 2-4 value is the average weekly clinically significant hypoglycemia episode rate 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 clinically significant hypoglycemic episodes during the 2-week baseline period.
Measured: Baseline, Week 2-4 (Treatment Period 1)
Total Amount of Gastric Carbohydrates Administered to Treat Hypoglycemia
Total amount of gastric carbohydrates administered via nasogastric tube or gastrostomy per week to treat hypoglycemia. The Week 2-4 value is the average weekly total amount of gastric carbohydrates across the last 3 weeks of treatment period. The change from Baseline to Week 2-4 is also provided. The Baseline value is the value recorded during the 2-week Baseline period.
Measured: Baseline, Week 2-4 (Treatment Period 1)
Rate of Gastric Carbohydrates Administrations to Treat Hypoglycemia
Rate of gastric carbohydrate administrations via nasogastric tube or gastrostomy per week to treat hypoglycemia, calculated as the number of times gastric carbohydrates were administered to treat hypoglycemia per week. The Week 2-4 value is the average weekly rate of gastric carbohydrate administrations 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 value recorded during the 2-week Baseline period.
Measured: Baseline, Week 2-4 (Treatment Period 1)
Extent of Hypoglycemia
Extent of hypoglycemia (area over the glucose curve [AOCglucose] below 70 mg/dL [3.9 mmol/L]) and below 54 mg/dL [3.0 mmol/L] as measured by continuous glucose monitoring. The extent of hypoglycemia was defined as the AOCglucose under the threshold divided by total duration in hours of CGM measurement. A reduction in this measure indicates a benefit. The Week 2-4 value is the average weekly extent of hypoglycemia below 70 mg/dL and below 54 mg/dL 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 value assessed during the 14 days before randomization.
Measured: Baseline, Week 2-4 (Treatment Period 1)
Amount of Nightly Gastric Carbohydrates Administered
Amount of nightly (midnight to 6 am) gastric carbohydrates administered via nasogastric tube or gastrostomy per week. The change from Baseline is also provided for each week. The Baseline value is the value recorded during the 2-week Baseline period.
Measured: Baseline, Week 2-4 (Treatment Period 1)
Total Amount of Gastric Carbohydrates Administered
Total amount of gastric carbohydrates administered (regardless of whether this was to treat hypoglycemia or not) via nasogastric tube or gastrostomy per week. The Week 2-4 value is the average amount of gastric carbohydrates administered via nasogastric tube or gastrostomy 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 value recorded during the 2-week baseline period.
Measured: Baseline, Week 2-4 (Treatment Period 1)
Percent Time in Hypoglycemia
Percent time in hypoglycemia (PG <70 mg/dL [3.9 mmol/L]) as measured by continuous glucose monitoring. The Week 2-4 value is the average weekly percent of time in hypoglycemia 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 value assessed during the 14 days before randomization.
Measured: Baseline, Week 2-4 (Treatment Period 1)
Rate of Hypoglycemic Episodes
Rate of hypoglycemic episodes, defined as number of episodes with PG <70 mg/dL (3.9 mmol/L) for 15 minutes or more per week, as measured by continuous glucose monitoring. 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 14 days before randomization.
Measured: Baseline, Week 2-4 (Treatment Period 1)
Percent Time in Hypoglycemia in Treatment Period 2
Percent time in hypoglycemia (PG <70 mg/dL [3.9 mmol/L]) as measured by continuous glucose monitoring. The Week 6-8 value is the average weekly percent of time in hypoglycemia across the last 3 weeks of the treatment period. The change from Baseline to Week 6-8 is also provided. The Baseline value is the value assessed during the 14 days before randomization.
Measured: Baseline, Week 6-8 (Treatment Period 2)
Rate of Gastric Carbohydrates Administrations to Treat Hypoglycemia in Treatment Period 2
Rate of gastric carbohydrate administrations via nasogastric tube or gastrostomy per week to treat hypoglycemia, calculated as the number of times gastric carbohydrates were administered to treat hypoglycemia per week. The Week 6-8 value is the average weekly rate of gastric carbohydrate administrations across the last 3 weeks of the treatment period. The change from Baseline to Week 6-8 is also provided. The Baseline value is the value recorded during the 2-week Baseline period.
Measured: Baseline, Week 6-8 (Treatment Period 2)
Rate of Hypoglycemic Episodes in Treatment Period 2
Hypoglycemia episode rate, defined as average weekly number of hypoglycemic episodes (PG <70 mg/dL [3.9 mmol/L]) as detected by SMPG. The Week 6-8 value is the average weekly number of hypoglycemic episodes across the last 3 weeks of the treatment period. The change from Baseline to Week 6-8 is also provided. The Baseline value is the average weekly number of hypoglycemic episodes during the 2-week baseline period.
Measured: Baseline, Week 6-8 (Treatment Period 2)
Rate of Clinically Significant Hypoglycemia Episodes in Treatment Period 2
Change from Baseline to Week 6-8 in the clinically significant (CS) hypoglycemia episode rate in treatment period 2, defined as the average weekly number of episodes <54 mg/dL (3.0 mmol/L), for 15 minutes or more, as measured by continuous glucose monitoring (CGM). The Week 6-8 value is the average weekly CS hypoglycemia episode rate across the last 3 weeks of the treatment period. The Baseline value is the average weekly number of CS hypoglycemic episodes during the 14 days before randomization.
Measured: Baseline, Week 6-8 (Treatment Period 2)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2018
- Study start7 Feb 2019
- Primary completion31 Aug 2020
- Study completion5 Oct 2020
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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zealand Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Charlotte Teglman Schioeler, Study director, Zealand Pharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03777176
- Sponsor's protocol ID
- ZP4207-17109
- Status last verified
- Nov 2023
- First submitted
- 13 Dec 2018
- Last updated
- 13 Dec 2023
- 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 13 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism
- ClinicalTrials.gov ID
- NCT03777176
- Acronym / sponsor ID
- ZP4207-17109
- Conditions
- Congenital Hyperinsulinism
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Zealand PharmaIndustry (pharma, biotech, devices)
- Study start
- 7 Feb 2019 (actual)
- Primary completion
- 31 Aug 2020 (actual)
- Study completion
- 5 Oct 2020 (actual)
- First posted
- 17 Dec 2018 (actual)
- Results
- Posted 13 Dec 2023
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 and safety of dasiglucagon administered as a subcutaneous (SC) infusion in reducing hypoglycemia in children with CHI.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Standard of Care + dasiglucagon | Experimental | Drug: Dasiglucagon, Other: Standard of Care4 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 |
| Standard of Care Only | Other | Drug: Dasiglucagon, Other: Standard of Care4 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 |
Who may qualify
- Ages
- 3 months to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (13 listed)
- 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
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 analog | Drug | Standard of Care + dasiglucagon, Standard of Care Only |
| Standard of CareStandard of care according to site and/or country | Other | Standard of Care + dasiglucagon, Standard of Care Only |
Primary outcome
Hypoglycemia Episode Rate
Time frame: Baseline, Week 2-4 (Treatment Period 1)
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.
14 secondary outcomes
Increase in Fasting Tolerance
Time frame: Baseline, Week 2-4 (Treatment Period 1)
Percent Time in Range 70-180 mg/dL
Time frame: Baseline, Week 2-4 (Treatment Period 1)
Clinically Significant Hypoglycemia Episode Rates
Time frame: Baseline, Week 2-4 (Treatment Period 1)
Total Amount of Gastric Carbohydrates Administered to Treat Hypoglycemia
Time frame: Baseline, Week 2-4 (Treatment Period 1)
Rate of Gastric Carbohydrates Administrations to Treat Hypoglycemia
Time frame: Baseline, Week 2-4 (Treatment Period 1)
Extent of Hypoglycemia
Time frame: Baseline, Week 2-4 (Treatment Period 1)
Amount of Nightly Gastric Carbohydrates Administered
Time frame: Baseline, Week 2-4 (Treatment Period 1)
Total Amount of Gastric Carbohydrates Administered
Time frame: Baseline, Week 2-4 (Treatment Period 1)
Percent Time in Hypoglycemia
Time frame: Baseline, Week 2-4 (Treatment Period 1)
Rate of Hypoglycemic Episodes
Time frame: Baseline, Week 2-4 (Treatment Period 1)
Percent Time in Hypoglycemia in Treatment Period 2
Time frame: Baseline, Week 6-8 (Treatment Period 2)
Rate of Gastric Carbohydrates Administrations to Treat Hypoglycemia in Treatment Period 2
Time frame: Baseline, Week 6-8 (Treatment Period 2)
Rate of Hypoglycemic Episodes in Treatment Period 2
Time frame: Baseline, Week 6-8 (Treatment Period 2)
Rate of Clinically Significant Hypoglycemia Episodes in Treatment Period 2
Time frame: Baseline, Week 6-8 (Treatment Period 2)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Dec 2023.
- 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.
Charlotte Teglman Schioeler study director
Zealand Pharma
10 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Dasiglucagon in people with Congenital Hyperinsulinism, run by Zealand Pharma at 10 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 4 inclusion and 13 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 13 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United Kingdom, United States, Germany, Israel. 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 phase mean?
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 13 Dec 2023). 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?
Hypoglycemia Episode Rate (time frame: Baseline, Week 2-4 (Treatment Period 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 13 Dec 2023.