Congenital HyperinsulinismPhase 2NCT02937558
CSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism
A Phase 2 study testing Glucagon in people with Congenital Hyperinsulinism, run by Xeris Pharmaceuticals at 5 sites in United States.
Trial summary
This is a Phase 2, multi-center, randomized, placebo-controlled, double-blind trial with open-label follow-up designed to assess the efficacy of Xeris Glucagon delivered as a continuous subcutaneous infusion to prevent hypoglycemia with lower intravenous glucose infusion rates in children < 1 year of age with… Read more about this study
- Who is it for?
- Ages Up to 12 months
- Conditions the study is for
- Congenital Hyperinsulinism
- What is being tested?
- Glucagon
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 5 participants
- Where
- 5 sitesUnited States
- Study dates
- Oct 2016to Oct 2018
- Phase
- Phase 2
- 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 Up to 12 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosed with hyperinsulinism:
- Biochemical; detectable insulin (i.e., ≥1 µIU/L) at time of hypoglycemia (i.e, blood glucose <50 mg/dl), and/or suppressed free fatty acids (FFA), and/or suppressed beta-hydroxybutyrate (BOHB) and/or glycemic response to glucagon at time of hypoglycemia.
- Absolute necessity of intravenous glucose to prevent hypoglycemia:
- Having failed diazoxide therapy as defined by inadequacy of 5 days maximum dose of diazoxide to eliminate the need for IV glucose, not necessarily that diazoxide has no effect.
- May be on diazoxide and/or octreotide, but these drugs will be weaned off prior to randomization.
- May be on dextrose feeds.
- Patient may be a participant in other study protocols such as observational studies, as long as no investigational intervention has taken place within 24 hrs. prior to screening.
- Less than 12 months of age at screening.
Who cannot join
- History of allergy to glucagon or excipients in the CSI-Glucagon formulation.
- Currently receiving, or less than 12 hours removed from IV glucagon treatment that resulted in a best achievable GIR > 8 mg/(kg*min), prior to the start of study drug.
- Diazoxide naïve or within five days of starting diazoxide.
- Receiving steroids at doses larger than 20 mg/m2/day (hydrocortisone equivalent).
- Patients with sepsis.
- Receiving alpha or beta agonists for blood pressure support.
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- Received an investigational or other study drug within 5 half-lives of drug.
- Body weight less than or equal to 2.3 kg/5.0 lbs.
- History of pancreatectomy and GIR < 8 mg/(kg*min) after weaning of all concomitant therapies.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Cook Children's Medical Center
Fort Worth, Texas, 76104
Baylor College of Medicine, Texas Children's Hospital
Houston, Texas, 77030
UCLA Mattel Children's Hospital
Los Angeles, California, 90095
UCSF School of Medicine, Division of Pediatric Endocrinology
San Francisco, California, 94143
Washington University, St. Louis Children's Hospital
St Louis, Missouri, 63130
About this study
Official title: A Phase 2 Proof-of-Concept Study of CSI-Glucagon™ (Continuous Subcutaneous Glucagon Infusion) to Prevent Hypoglycemia With Lower Intravenous Glucose Infusion Rates in Children up to One Year of Age With Congenital Hyperinsulinism
This is a Phase 2, multi-center, randomized, placebo-controlled, double-blind trial with open-label follow-up designed to assess the efficacy of Xeris Glucagon delivered as a continuous subcutaneous infusion to prevent hypoglycemia with lower intravenous glucose infusion rates in children < 1 year of age with congenital hyperinsulinism.
Read the detailed description
This is a Phase 2, multi-center, randomized, placebo-controlled, double-blind (DB) parallel group study with open-label follow-up designed to evaluate the efficacy of CSI-Glucagon™ for the prevention of hypoglycemia with lower IV glucose infusion rates when delivered subcutaneously to patients up to 1 year of age with congenital hyperinsulinism. CSI-Glucagon™ is expected to provide a better inpatient treatment option compared to the current standard of care.
The study will consist of three phases:
- Baseline Phase: First is a baseline stabilization phase during which concomitant therapy with octreotide and diazoxide will be safely weaned and continuous enteric feed will be held constant to the degree possible, with the only factors varying being meal size and IV glucose infusion rate (GIR) adjusted by a set plasma glucose measurement driven algorithm.
- Blinded, Randomized Treatment Phase: Following the stabilization phase, subjects will be randomly assigned to blinded treatment with either glucagon or placebo, which will be delivered for up to 48 hours with an OmniPod® infusion pump with the controller set to a starting basal rate for glucagon of 5 μg/kg/hr and GIR adjustments used to maintain euglycemia. After 48 hours of blinded treatment, all subjects will transition to open-label active treatment. However, if GIR reduction from baseline is < 20% at 24 hours, subjects will be transitioned early to the open-label phase.
- Open-label Treatment Phase: The third study period will involve use of CSI-Glucagon™ to manage blood glucose with minimal GIR for up to 28 days of cumulative exposure.
- Conditions
- Congenital Hyperinsulinism
- Being tested
- Glucagon, Placebo
- Keywords
- hypoglycemia
- Medical subject headings
- Congenital Hyperinsulinism, Hypoglycemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GlucagonDrug
Also called CSI-Glucagon (continuous subcutaneous glucagon infusion)
Room-temperature-stable, non-aqueous injectable liquid formulation of synthetic glucagon peptide
PlaceboOther
Isotonic saline
| Study group | Role | What they receive |
|---|---|---|
| CSI-Glucagon (Double-Blind Phase - 2 days) | Experimental | Glucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr. Receives: Drug: Glucagon |
| Placebo (Double-Blind Phase - 2 days) | Placebo comparator | Vehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump. Receives: Other: Placebo |
| CSI-Glucagon (Open-label Phase - Up to 28 days) | Experimental | Glucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr. Receives: Drug: Glucagon |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Double-Blind)
Change from baseline in glucose infusion rate (GIR) will be determined for each subject at 24 and 48 hours from the start of blinded treatment. Subjects with a decrease in GIR ≥ 20% at 24 hours, and ≥ 33% at 48 hours will be considered to have had a clinically meaningful treatment response.
Measured: Baseline to end of blinded treatment at 24 or 48 hours
Secondary outcomes (3)
Percent Change in GIR (Double-Blind)
The groups will be compared for mean percent change in GIR from baseline to the end of the double-blind study phase.
Measured: Baseline to the end of blinded treatment at 24 or 48 hours
Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Open-Label)
Change from baseline in glucose infusion rate (GIR) will be determined for each subject at the end of open-label treatment. Subjects with a decrease in GIR ≥ 33% will be considered to have had a clinically meaningful treatment response.
Measured: Baseline to the end of open-label treatment at 72 hours
Percent Change in Glucose Infusion Rate (Open-Label)
The groups will be compared for mean percent change in GIR from baseline to the end of the open-label study phase.
Measured: Baseline to end of treatment at 72 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2016 (estimated)
- Study startOct 2016
- Primary completionSep 2018
- Study completionOct 2018
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
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Xeris Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02937558
- Sponsor's protocol ID
- XSGO-CH01
- Other IDs
- 1R44DK105691-01
- Status last verified
- Nov 2019
- First submitted
- 14 Oct 2016
- Last updated
- 10 Dec 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Proof-of-Concept Study of CSI-Glucagon™ (Continuous Subcutaneous Glucagon Infusion) to Prevent Hypoglycemia With Lower Intravenous Glucose Infusion Rates in Children up to One Year of Age With Congenital Hyperinsulinism
- ClinicalTrials.gov ID
- NCT02937558
- Acronym / sponsor ID
- XSGO-CH01
- Conditions
- Congenital Hyperinsulinism
- Keywords
- hypoglycemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Xeris PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- Oct 2016
- Primary completion
- Sep 2018 (actual)
- Study completion
- Oct 2018 (actual)
- First posted
- 18 Oct 2016 (estimated)
- Results
- Posted 10 Dec 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 2, multi-center, randomized, placebo-controlled, double-blind trial with open-label follow-up designed to assess the efficacy of Xeris Glucagon delivered as a continuous subcutaneous infusion to prevent hypoglycemia with lower intravenous glucose infusion rates in children < 1 year of age with congenital hyperinsulinism.
Detailed description
This is a Phase 2, multi-center, randomized, placebo-controlled, double-blind (DB) parallel group study with open-label follow-up designed to evaluate the efficacy of CSI-Glucagon™ for the prevention of hypoglycemia with lower IV glucose infusion rates when delivered subcutaneously to patients up to 1 year of age with congenital hyperinsulinism. CSI-Glucagon™ is expected to provide a better inpatient treatment option compared to the current standard of care.
The study will consist of three phases:
- Baseline Phase: First is a baseline stabilization phase during which concomitant therapy with octreotide and diazoxide will be safely weaned and continuous enteric feed will be held constant to the degree possible, with the only factors varying being meal size and IV glucose infusion rate (GIR) adjusted by a set plasma glucose measurement driven algorithm.
- Blinded, Randomized Treatment Phase: Following the stabilization phase, subjects will be randomly assigned to blinded treatment with either glucagon or placebo, which will be delivered for up to 48 hours with an OmniPod® infusion pump with the controller set to a starting basal rate for glucagon of 5 μg/kg/hr and GIR adjustments used to maintain euglycemia. After 48 hours of blinded treatment, all subjects will transition to open-label active treatment. However, if GIR reduction from baseline is < 20% at 24 hours, subjects will be transitioned early to the open-label phase.
- Open-label Treatment Phase: The third study period will involve use of CSI-Glucagon™ to manage blood glucose with minimal GIR for up to 28 days of cumulative exposure.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| CSI-Glucagon (Double-Blind Phase - 2 days) | Experimental | Drug: GlucagonGlucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr. |
| Placebo (Double-Blind Phase - 2 days) | Placebo comparator | Other: PlaceboVehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump. |
| CSI-Glucagon (Open-label Phase - Up to 28 days) | Experimental | Drug: GlucagonGlucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr. |
Who may qualify
- Ages
- Up to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Diagnosed with hyperinsulinism:
- Absolute necessity of intravenous glucose to prevent hypoglycemia:
- Patient may be a participant in other study protocols such as observational studies, as long as no investigational intervention has taken place within 24 hrs. prior to screening.
- Less than 12 months of age at screening.
Key exclusion criteria (9 listed)
- History of allergy to glucagon or excipients in the CSI-Glucagon formulation.
- Currently receiving, or less than 12 hours removed from IV glucagon treatment that resulted in a best achievable GIR > 8 mg/(kg*min), prior to the start of study drug.
- Diazoxide naïve or within five days of starting diazoxide.
- Receiving steroids at doses larger than 20 mg/m2/day (hydrocortisone equivalent).
- Patients with sepsis.
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 |
|---|---|---|
| GlucagonAlso: CSI-Glucagon (continuous subcutaneous glucagon infusion)Room-temperature-stable, non-aqueous injectable liquid formulation of synthetic glucagon peptide | Drug | CSI-Glucagon (Double-Blind Phase - 2 days), CSI-Glucagon (Open-label Phase - Up to 28 days) |
| PlaceboIsotonic saline | Other | Placebo (Double-Blind Phase - 2 days) |
Primary outcome
Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Double-Blind)
Time frame: Baseline to end of blinded treatment at 24 or 48 hours
Change from baseline in glucose infusion rate (GIR) will be determined for each subject at 24 and 48 hours from the start of blinded treatment. Subjects with a decrease in GIR ≥ 20% at 24 hours, and ≥ 33% at 48 hours will be considered to have had a clinically meaningful treatment response.
3 secondary outcomes
Percent Change in GIR (Double-Blind)
Time frame: Baseline to the end of blinded treatment at 24 or 48 hours
Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Open-Label)
Time frame: Baseline to the end of open-label treatment at 72 hours
Percent Change in Glucose Infusion Rate (Open-Label)
Time frame: Baseline to end of treatment at 72 hours
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Dec 2019.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Glucagon in people with Congenital Hyperinsulinism, run by Xeris Pharmaceuticals at 5 sites in United States.
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 9 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 10 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 10 Dec 2019). 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?
Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Double-Blind) (time frame: Baseline to end of blinded treatment at 24 or 48 hours). 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 10 Dec 2019; AzTrials last refreshed its copy on 8 Nov 2026.