Primary outcomeCount of participants· Baseline to end of blinded treatment at 24 or 48 hours
Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Double-Blind)
| Group | Value (Participants) |
|---|---|
| CSI-Glucagon | 2 |
| Placebo | 0 |
Trial · Interventional
CompletedPhase 2Results postedRegistry dataCSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism
Overview
- Status
- Completed
- Phase
- Phase 2
- Enrollment
- 5 participants (actual)
- Sponsor
- Xeris Pharmaceuticals · Industry
- Design
- Randomized · Quadruple masking
- Countries
1 country
United States
- Start
- Oct 2016
- Primary completion
- Sept 2018
- Completion
- Oct 2018
- First posted
- 18 Oct 2016 (estimated)
- Last update posted
- 10 Dec 2019
- Conditions
- Congenital Hyperinsulinism
Timeline
1 trial · 1 completed · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
2016201720182019
- Ph 2
CSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism
NCT02937558Oct 2016 to Oct 2018
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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.
Interventions
- GlucagonDrug
- PlaceboOther
Arms3
Experimental
CSI-Glucagon (Double-Blind Phase - 2 days)
Glucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr.
Placebo comparator
Placebo (Double-Blind Phase - 2 days)
Vehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump.
Experimental
CSI-Glucagon (Open-label Phase - Up to 28 days)
Glucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr.
Primary outcomes1
Outcomes the sponsor listed as primary.
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
Registry description
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.
Secondary outcomes(3)
Percent Change in GIR (Double-Blind)
Time frame · Baseline to the end of blinded treatment at 24 or 48 hours
Registry description
The groups will be compared for mean percent change in GIR from baseline to the end of the double-blind study phase.
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
Registry description
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.
Percent Change in Glucose Infusion Rate (Open-Label)
Time frame · Baseline to end of treatment at 72 hours
Registry description
The groups will be compared for mean percent change in GIR from baseline to the end of the open-label study phase.
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Secondary and other outcome results(3)
Secondary outcomeMean· Baseline to the end of blinded treatment at 24 or 48 hours
Percent Change in GIR (Double-Blind)
| Group | Value (% change) | Spread |
|---|---|---|
| CSI-Glucagon | -50.1 | 4.5 |
| Placebo | 1.9 | 45.9 |
Secondary outcomeCount of participants· Baseline to the end of open-label treatment at 72 hours
Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Open-Label)
| Group | Value (Participants) |
|---|---|
| CSI-Glucagon | 4 |
| Placebo | 0 |
Secondary outcomeMean· Baseline to end of treatment at 72 hours
Percent Change in Glucose Infusion Rate (Open-Label)
| Group | Value (% change) | Spread |
|---|---|---|
| CSI-Glucagon | -51.2 | 10.3 |
| Placebo | not posted | – |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| CSI-Glucagon (Double-Blind)Glucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr. Glucagon: Room-temperature-stable, non-aqueous injectable liquid formulation of synthetic glucagon peptide | 0 / 3 | 0 / 3 | 0 / 3 |
| PlaceboVehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump. Placebo: Isotonic saline | 0 / 2 | 0 / 2 | 0 / 2 |
| CSI-Glucagon (Open-Label)Glucagon solution delivered as a continuous subcutaneous infusion via a patch pump at a starting dosage of 5 mcg/kg/hr. Glucagon: Room-temperature-stable, non-aqueous injectable liquid formulation of synthetic glucagon peptide | 0 / 5 | 0 / 5 | 0 / 5 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- up to 12 Months
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria:
1. Diagnosed with hyperinsulinism:
a. 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.
2. Absolute necessity of intravenous glucose to prevent hypoglycemia:
1. 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.
2. May be on diazoxide and/or octreotide, but these drugs will be weaned off prior to randomization.
3. May be on dextrose feeds.
3. 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.
4. Less than 12 months of age at screening.
Exclusion Criteria:
1. History of allergy to glucagon or excipients in the CSI-Glucagon formulation.
2. 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.
3. Diazoxide naïve or within five days of starting diazoxide.
4. Receiving steroids at doses larger than 20 mg/m2/day (hydrocortisone equivalent).
5. Patients with sepsis.
6. Receiving alpha or beta agonists for blood pressure support.
7. Received an investigational or other study drug within 5 half-lives of drug.
8. Body weight less than or equal to 2.3 kg/5.0 lbs.
9. History of pancreatectomy and GIR \< 8 mg/(kg\*min) after weaning of all concomitant therapies.
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