Trial Outcomes & Findings for Effect of Exendin-(9-39) on Glycemic Control in Subjects With Congenital Hyperinsulinism (NCT NCT00571324)
NCT ID: NCT00571324
Last Updated: 2017-12-11
Results Overview
To examine the effect of Exendin-(9-39) on fasting blood glucose levels, samples were collected at various time points before and during the infusion \[Exendin-(9-39) or vehicle\] including: 60 minutes before the start of the infusion, again at the start of the infusion (Time 0), and then every 20 minutes until 6 hours after the start of the infusion. Using this information, the mean blood glucose area under the curve (AUC) from the start of the infusion to the end of the infusion (360 minutes) was calculated for each both Exendin-(9-39) and vehicle and compared.
COMPLETED
PHASE1/PHASE2
9 participants
6 hours
2017-12-11
Participant Flow
Subjects with confirmed genetic and clinical diagnosis of Adenosine triphosphate (ATP)-sensitive potassium channels (KATP) hyperinsulinism were recruited from the Hyperinsulinism Center at the Children's Hospital of Philadelphia
Participant milestones
| Measure |
Exendin-(9-39) First, the Vehicle
Exendin-(9-39) was administered intravenously (IV) after an overnight fast. Exendin-(9-39) was infused over 6 hours with the dose slowly escalating from 100pmol/kg/min for 2 hours, then 300pmol/kg/min for another 2 hours followed by 500pmol/kg/min for the last 2 hours of the infusion. The following day, after another overnight fast, normal saline (control) vehicle infusion was administered intravenously (IV) over 6 hours. During both infusions, blood glucose levels were measured every 20 minutes.
|
Vehicle First, Then Exendin-(9-39)
Normal saline vehicle infusion was administered intravenously (IV) after an overnight fast. The infusion was given over 6 hours. The following day, after another overnight fast, Exendin-(9-39) was infused over 6 hours with the dose slowly escalating from 100pmol/kg/min for 2 hours, then 300pmol/kg/min for another 2 hours followed by 500pmol/kg/min for the last 2 hours of the infusion. During both infusions, blood glucose levels were measured every 20 minutes.
|
|---|---|---|
|
Intervention 1 (7 Hours)
STARTED
|
5
|
4
|
|
Intervention 1 (7 Hours)
COMPLETED
|
5
|
4
|
|
Intervention 1 (7 Hours)
NOT COMPLETED
|
0
|
0
|
|
Intervention 2 (7 Hours)
STARTED
|
5
|
4
|
|
Intervention 2 (7 Hours)
COMPLETED
|
5
|
4
|
|
Intervention 2 (7 Hours)
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Effect of Exendin-(9-39) on Glycemic Control in Subjects With Congenital Hyperinsulinism
Baseline characteristics by cohort
| Measure |
Subject Population
n=9 Participants
All subjects enrolled and treated in the protocol served as their own control. Because of this and the small sample size, baseline characteristic data is presented together.
|
|---|---|
|
Age, Categorical
<=18 years
|
4 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=5 Participants
|
|
Mutation (ABCC8)
Heterozygous dominant ABCC8 mutations
|
6 participants
n=5 Participants
|
|
Mutation (ABCC8)
Homozygous common recessive Ashkenazi mutation
|
2 participants
n=5 Participants
|
|
Mutation (ABCC8)
Compound heterozygous recessive ABCC8 mutations
|
1 participants
n=5 Participants
|
|
Pancreatectomy
None
|
6 participants
n=5 Participants
|
|
Pancreatectomy
85% Pancreatectomy
|
1 participants
n=5 Participants
|
|
Pancreatectomy
95% Pancreatectomy
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 hoursPopulation: Subjects served as their own control for comparison between effects of Exendin-(9-39) and the normal saline vehicle.
To examine the effect of Exendin-(9-39) on fasting blood glucose levels, samples were collected at various time points before and during the infusion \[Exendin-(9-39) or vehicle\] including: 60 minutes before the start of the infusion, again at the start of the infusion (Time 0), and then every 20 minutes until 6 hours after the start of the infusion. Using this information, the mean blood glucose area under the curve (AUC) from the start of the infusion to the end of the infusion (360 minutes) was calculated for each both Exendin-(9-39) and vehicle and compared.
Outcome measures
| Measure |
Exendin-(9-39)
n=9 Participants
Exendin-(9-39) was administered intravenously (IV) after an overnight fast. Exendin-(9-39) was infused over 6 hours with the dose slowly escalating from 100pmol/kg/min for 2 hours, then 300pmol/kg/min for another 2 hours followed by 500pmol/kg/min for the last 2 hours of the infusion.
|
Vehicle
n=9 Participants
Normal saline vehicle infusion was administered intravenously (IV) after an overnight fast. The infusion was given over 6 hours.
|
|---|---|---|
|
Mean Blood Glucose Area Under the Curve (AUC 0-6h)
|
2,096 mmol*min/L
Standard Error 454
|
1,678 mmol*min/L
Standard Error 281
|
SECONDARY outcome
Timeframe: 6 hoursPopulation: Subjects served as their own control for comparison between effects of Exendin-(9-39) and the normal saline vehicle.
To examine the effect of Exendin-(9-39) on plasma insulin levels, samples were collected at various time points before and during the infusion \[Exendin-(9-39) or vehicle\] including: 60 minutes before the start of the infusion, again at the start of the infusion (Time 0), and then every 20 minutes until 6 hours after the start of the infusion. Using this information, the mean plasma insulin area under the curve (AUC) from the start of the infusion to the end of the infusion (360 minutes) was calculated for each both Exendin-(9-39) and vehicle and compared.
Outcome measures
| Measure |
Exendin-(9-39)
n=9 Participants
Exendin-(9-39) was administered intravenously (IV) after an overnight fast. Exendin-(9-39) was infused over 6 hours with the dose slowly escalating from 100pmol/kg/min for 2 hours, then 300pmol/kg/min for another 2 hours followed by 500pmol/kg/min for the last 2 hours of the infusion.
|
Vehicle
n=9 Participants
Normal saline vehicle infusion was administered intravenously (IV) after an overnight fast. The infusion was given over 6 hours.
|
|---|---|---|
|
Mean Plasma Insulin Area Under the Curve (AUC 0-6h)
|
21,016 pmol*min/L
Standard Error 7,410
|
25,842 pmol*min/L
Standard Error 12,397
|
SECONDARY outcome
Timeframe: 6 hoursPopulation: Subjects served as their own control for comparison between effects of Exendin-(9-39) and the normal saline vehicle.
To examine the effect of Exendin-(9-39) on plasma glucagon levels, samples were collected at various time points before and during the infusion \[Exendin-(9-39) or vehicle\] including: 60 minutes before the start of the infusion, again at the start of the infusion (Time 0), and then every 20 minutes until 6 hours after the start of the infusion. Using this information, the mean plasma glucagon area under the curve (AUC) from the start of the infusion to the end of the infusion (360 minutes) was calculated for each both Exendin-(9-39) and vehicle and compared.
Outcome measures
| Measure |
Exendin-(9-39)
n=9 Participants
Exendin-(9-39) was administered intravenously (IV) after an overnight fast. Exendin-(9-39) was infused over 6 hours with the dose slowly escalating from 100pmol/kg/min for 2 hours, then 300pmol/kg/min for another 2 hours followed by 500pmol/kg/min for the last 2 hours of the infusion.
|
Vehicle
n=9 Participants
Normal saline vehicle infusion was administered intravenously (IV) after an overnight fast. The infusion was given over 6 hours.
|
|---|---|---|
|
Mean Plasma Glucagon Area Under the Curve (AUC 0-6h)
|
21,243 pg*min/dL
Standard Error 5,836
|
21,867 pg*min/dL
Standard Error 4,432
|
SECONDARY outcome
Timeframe: 6 hoursPopulation: Subjects served as their own control for comparison between effects of Exendin-(9-39) and the normal saline vehicle.
To examine the effect of Exendin-(9-39) on plasma intact glucagon-like Peptide-1 (GLP-1) levels, samples were collected at various time points before and during the infusion \[Exendin-(9-39) or vehicle\] including: 60 minutes before the start of the infusion, again at the start of the infusion (Time 0), and then every 20 minutes until 6 hours after the start of the infusion. Using this information, the mean intact GLP-1 area under the curve (AUC) from the start of the infusion to the end of the infusion (360 minutes) was calculated for each both Exendin-(9-39) and vehicle and compared.
Outcome measures
| Measure |
Exendin-(9-39)
n=9 Participants
Exendin-(9-39) was administered intravenously (IV) after an overnight fast. Exendin-(9-39) was infused over 6 hours with the dose slowly escalating from 100pmol/kg/min for 2 hours, then 300pmol/kg/min for another 2 hours followed by 500pmol/kg/min for the last 2 hours of the infusion.
|
Vehicle
n=9 Participants
Normal saline vehicle infusion was administered intravenously (IV) after an overnight fast. The infusion was given over 6 hours.
|
|---|---|---|
|
Mean Plasma Intact Glucagon-Like Peptide-1 (GLP-1) Area Under the Curve (AUC 0-6h)
|
1,970 pmol*min/L
Standard Error 1,284
|
1,992 pmol*min/L
Standard Error 1,203
|
Adverse Events
Exendin-(9-39)
Vehicle
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Exendin-(9-39)
n=9 participants at risk
Exendin-(9-39) was administered intravenously (IV) after an overnight fast. Exendin-(9-39) was infused over 6 hours with the dose slowly escalating from 100pmol/kg/min for 2 hours, then 300pmol/kg/min for another 2 hours followed by 500pmol/kg/min for the last 2 hours of the infusion.
|
Vehicle
n=9 participants at risk
Normal saline vehicle infusion was administered intravenously (IV) after an overnight fast. The infusion was given over 6 hours.
|
|---|---|---|
|
Endocrine disorders
Hypoglycemia
|
0.00%
0/9 • 30 days following the last administration of study treatment (either Exendin-(9-39) or vehicle)
|
33.3%
3/9 • Number of events 3 • 30 days following the last administration of study treatment (either Exendin-(9-39) or vehicle)
|
|
General disorders
Headache
|
0.00%
0/9 • 30 days following the last administration of study treatment (either Exendin-(9-39) or vehicle)
|
11.1%
1/9 • Number of events 1 • 30 days following the last administration of study treatment (either Exendin-(9-39) or vehicle)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place