Trial Outcomes & Findings for Phase II Safety and Efficacy Study of 18FDOPA PET-CT in Children With Hyperinsulinemic Hypoglycemia (NCT NCT01468454)
NCT ID: NCT01468454
Last Updated: 2022-10-13
Results Overview
To determine the sensitivity and specificity of 18F-DOPA PET/CT for the detection of a focal pancreatic lesion in infants and children with poorly controlled hyperinsulinemic hypoglycemia.
COMPLETED
PHASE2
130 participants
Surgery typically occured within a week post PET
2022-10-13
Participant Flow
From January 2009 to July 2013, the study team recruited and enrolled 129 subjects referred to the Congenital Hyperinsulinism Center at the Children's Hospital of Philadelphia who were potential candidates for partial pancreatectomy surgery if focal lesions were suspected and were able to be localized.
1 subject was enrolled into the study (informed consent signed), however, was deemed ineligible due to subsequent additional clinical information provided after consent but prior to scheduled PET study. . A second subject had two scans, but is only counted once in the final analysis. This accounts for the enrollment discrepancy (130 vs.128 described in the protocol section)
Participant milestones
| Measure |
F-DOPA PET/CT Imaging vs Surgical Result
18 F-DOPA: 1 time injection of 0.08 - 0.16 mCi/kg of 18F-DOPA given 10-15 minutes prior image acquisition.
|
|---|---|
|
Overall Study
STARTED
|
128
|
|
Overall Study
PETs Completed
|
128
|
|
Overall Study
Had Both PET and Surgery
|
119
|
|
Overall Study
COMPLETED
|
119
|
|
Overall Study
NOT COMPLETED
|
9
|
Reasons for withdrawal
| Measure |
F-DOPA PET/CT Imaging vs Surgical Result
18 F-DOPA: 1 time injection of 0.08 - 0.16 mCi/kg of 18F-DOPA given 10-15 minutes prior image acquisition.
|
|---|---|
|
Overall Study
Did not have surgery after PET
|
9
|
Baseline Characteristics
Phase II Safety and Efficacy Study of 18FDOPA PET-CT in Children With Hyperinsulinemic Hypoglycemia
Baseline characteristics by cohort
| Measure |
(18F-DOPA) PET/CT Imaging vs Surgical Results
n=128 Participants
18 F-DOPA: 1 time injection of 0.08 - 0.16 mCi/kg of 18F-DOPA given 10-15 minutes prior image acquisition.
|
|---|---|
|
Age, Categorical
<=18 years
|
128 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
57 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
71 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
27 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
101 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
3 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
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
95 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
12 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
14 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
121 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
4 participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Israel
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Paraguay
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Surgery typically occured within a week post PETPopulation: A single F-DOPA PET/CT imaging study was performed and reported prior to surgery. This data includes surgical histopathology confirmed as focal or diffuse. 19 of the 119 participants with surgical results had atypical histopathology that did not fit the definition of either category and therefore are excluded from this analysis.
To determine the sensitivity and specificity of 18F-DOPA PET/CT for the detection of a focal pancreatic lesion in infants and children with poorly controlled hyperinsulinemic hypoglycemia.
Outcome measures
| Measure |
PET/CT Imaging vs Surgical Result
n=100 Participants
PET/CT imaging results in patients with surgical histologic confirmation.
|
|---|---|
|
Accuracy of 18F-DOPA PET/CT Scans to Detect Focal Lesions in Children With Congenital Hyperinsulinism
True Negatives (PET diffuse/surgery diffuse)
|
34 cases
|
|
Accuracy of 18F-DOPA PET/CT Scans to Detect Focal Lesions in Children With Congenital Hyperinsulinism
False negatives (PET diffuse/surgery focal):
|
10 cases
|
|
Accuracy of 18F-DOPA PET/CT Scans to Detect Focal Lesions in Children With Congenital Hyperinsulinism
False positives (PET focal/surgery diffuse):
|
4 cases
|
|
Accuracy of 18F-DOPA PET/CT Scans to Detect Focal Lesions in Children With Congenital Hyperinsulinism
True positives (PET focal/surgery focal):
|
52 cases
|
SECONDARY outcome
Timeframe: evaluated with 72 hours or prior to pancreatic surgery (if any)Population: all participants who had both PET and surgery
To further evaluate the safety of 18-labeled L-fluorodeoxyphenylalanine (18F-DOPA) PET/CT imaging in infants and children with congenital hyperinsulinism \- subjects are monitored clinically for any signs or symptoms of adverse events for 72 hours post PET. Adverse events are documented and followed to resolution
Outcome measures
| Measure |
PET/CT Imaging vs Surgical Result
n=119 Participants
PET/CT imaging results in patients with surgical histologic confirmation.
|
|---|---|
|
Safety of 18F-DOPA PET/CT Scan - Number of Participants With Adverse Events
|
0 Participants
|
Adverse Events
(18F-DOPA) PET/CT Imaging
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Lisa J. States, MD
Children's Hospital of Philadelphia - Department of Radiology
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place