Trial Outcomes & Findings for CSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism (NCT NCT02937558)
NCT ID: NCT02937558
Last Updated: 2019-12-10
Results Overview
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.
COMPLETED
PHASE2
5 participants
Baseline to end of blinded treatment at 24 or 48 hours
2019-12-10
Participant Flow
A total of 5 subjects were randomized to active or placebo treatment during the double-blind phase of the study. All subjects completing double-blind treatment were eligible for open-label in-patient treatment with CSI Glucagon.
The first enrolled subject was not deemed evaluable for responsive to glucagon based upon prior history of glucagon resistance, per investigator.
Participant milestones
| Measure |
CSI-Glucagon
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
|
Placebo
Vehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump.
Placebo: Isotonic saline
|
|---|---|---|
|
Double-blind Phase
STARTED
|
3
|
2
|
|
Double-blind Phase
COMPLETED
|
3
|
2
|
|
Double-blind Phase
NOT COMPLETED
|
0
|
0
|
|
Open-label Phase
STARTED
|
5
|
0
|
|
Open-label Phase
COMPLETED
|
5
|
0
|
|
Open-label Phase
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
CSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism
Baseline characteristics by cohort
| Measure |
CSI-Glucagon
n=3 Participants
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
|
Placebo
n=2 Participants
Vehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump.
Placebo: Isotonic saline
|
Total
n=5 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
25 days
STANDARD_DEVIATION 12.49 • n=5 Participants
|
78.5 days
STANDARD_DEVIATION 79.90 • n=7 Participants
|
46.4 days
STANDARD_DEVIATION 50.33 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to end of blinded treatment at 24 or 48 hoursPopulation: Evaluable subjects
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.
Outcome measures
| Measure |
CSI-Glucagon
n=2 Participants
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
|
Placebo
n=2 Participants
Vehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump.
Placebo: Isotonic saline
|
|---|---|---|
|
Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Double-Blind)
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline to the end of blinded treatment at 24 or 48 hoursPopulation: Evaluable subjects
The groups will be compared for mean percent change in GIR from baseline to the end of the double-blind study phase.
Outcome measures
| Measure |
CSI-Glucagon
n=2 Participants
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
|
Placebo
n=2 Participants
Vehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump.
Placebo: Isotonic saline
|
|---|---|---|
|
Percent Change in GIR (Double-Blind)
|
-50.1 % change
Standard Deviation 4.5
|
1.9 % change
Standard Deviation 45.9
|
SECONDARY outcome
Timeframe: Baseline to the end of open-label treatment at 72 hoursPopulation: Evaluable subjects
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.
Outcome measures
| Measure |
CSI-Glucagon
n=4 Participants
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
|
Placebo
Vehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump.
Placebo: Isotonic saline
|
|---|---|---|
|
Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Open-Label)
|
4 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline to end of treatment at 72 hoursPopulation: Evaluable subjects
The groups will be compared for mean percent change in GIR from baseline to the end of the open-label study phase.
Outcome measures
| Measure |
CSI-Glucagon
n=4 Participants
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
|
Placebo
Vehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump.
Placebo: Isotonic saline
|
|---|---|---|
|
Percent Change in Glucose Infusion Rate (Open-Label)
|
-51.2 % change
Standard Deviation 10.3
|
—
|
Adverse Events
CSI-Glucagon (Double-Blind)
Placebo
CSI-Glucagon (Open-Label)
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place