CHI, Congenital Hyperinsulinism, Hyperinsulinism, Persistent Hyperinsulinemic Hypoglycemia of Infancy, PHHI
Conditions
Keywords
Congenital Hyperinsulinism, Hyperinsulinism, Persistent Hyperinsulinemic Hypoglycemia of Infancy, CHI, PHHI, F-DOPA, L-fluoro-dihydroxyphenylalanine
Brief summary
Children with congenital hyperinsulinism (CHI) have low blood sugar, and some of these children may require surgery. In this study, researchers affiliated with the University of Pennsylvania will test how well a radioactive drug (called F-DOPA) can detect a form of hyperinsulinism that may be cured by surgery. Eligible participants in this study will have positron emission tomography (PET) scans with F-DOPA prior to surgery.
Detailed description
For children with congenital hyperinsulinism (CHI), low blood sugar is caused by cells in the pancreas that release too much insulin. Some children with CHI have these cells throughout their pancreas; others have them located in specific areas of the pancreas. Children who have them located in specific areas of the pancreas may be cured with surgery. F-DOPA is a radioactive drug that may go to these very cells. F-DOPA can also be used for positron emission tomography (or PET), an imaging technique used in nuclear medicine departments. In this study, researchers will test the possibility of using PET with F-DOPA in the diagnosis of children with hyperinsulinism.
Interventions
DRUGF-DOPA
0.08-0.16 mCi/kg once
RADIATIONPET scan
Sponsors
University of Pennsylvania
Children's Hospital of Philadelphia
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
DIAGNOSTIC
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No
Inclusion criteria
* Any age, but primarily infants 0-6 months. * Children with confirmed diagnosis of congenital hyperinsulinism.
Exclusion criteria
* Cases in which surgery will not be considered by parents or guardians.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism | up to 1 month post surgical intervention | Comparison of PET scan results with outcome of surgery and histopathology results confirmed as focal or diffuse |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Safety of F-DOPA PET | 72 hours maximum or prior to pancreatic surgery | Number of Participants with Adverse Events |
Countries
United States
Participant flow
Recruitment details
From December 2004 to December 2008, the study team recruited and enrolled 106 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.
Pre-assignment details
The participants newly diagnosed with hypersulinism required surgery for hyperinsulinism. A subset of patients who seemed to respond to medical therapy or had continued hyperinsulinism after surgery were also included. All subjects were consented for PET scan and were eligible for surgery. 105 patients underwent F-DOPA PET with the same research procedures.
Participants by arm
| Arm | Count |
|---|---|
| Subjects Who Consented These demographics include all subject recruited who consented whether they are evaluable or not | 106 |
| Total | 106 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | consented but not scanned (Withdrawn due to genetics) | 1 |
| Overall Study | did not have surgery, so unevaluable | 7 |
| Overall Study | had atypical histology or had second PET scan after initial surgery - not counted twice | 3 |
Baseline characteristics
| Characteristic | Subjects Who Consented |
|---|---|
| Age, Categorical <=18 years | 106 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 19 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 87 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 4 Participants |
| Race (NIH/OMB) Black or African American | 6 Participants |
| Race (NIH/OMB) More than one race | 23 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 73 Participants |
| Region of Enrollment Brazil | 1 Participants |
| Region of Enrollment Canada | 7 Participants |
| Region of Enrollment Curacao | 1 Participants |
| Region of Enrollment Iran | 1 Participants |
| Region of Enrollment Israel | 1 Participants |
| Region of Enrollment Saudi Arabia | 1 Participants |
| Region of Enrollment United States | 94 Participants |
| Sex: Female, Male Female | 53 Participants |
| Sex: Female, Male Male | 53 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 105 |
| other Total, other adverse events | 0 / 105 |
| serious Total, serious adverse events | 0 / 105 |
Outcome results
Primary
The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism
Comparison of PET scan results with outcome of surgery and histopathology results confirmed as focal or diffuse
Time frame: up to 1 month post surgical intervention
Population: An F-DOPA PET/CT imaging study was performed and reported prior to surgery. This data includes surgical histopathology confirmed as focal or diffuse. 95 of the 106 original consented participants had surgical results that were evaluable. (1 consented but never scanned, 7 did not have surgery, 3 had surgery but were excluded for atypical histology that did not fit classic focal or diffuse or had second PET scan after initial surgery therefore not counted twice).
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Subjects Who Had PET and Surgery | The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism | True Negatives (PET diffuse/surgery diffuse) | 40 Participants |
| Subjects Who Had PET and Surgery | The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism | False negatives (PET diffuse/surgery focal): | 6 Participants |
| Subjects Who Had PET and Surgery | The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism | False positives (PET focal/surgery diffuse): | 3 Participants |
| Subjects Who Had PET and Surgery | The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism | True positives (PET focal/surgery focal): | 46 Participants |
95% CI: [80.9, 98.5]
95% CI: [76.6, 95.6]
Secondary
Safety of F-DOPA PET
Number of Participants with Adverse Events
Time frame: 72 hours maximum or prior to pancreatic surgery
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Subjects Who Had PET and Surgery | Safety of F-DOPA PET | 0 Participants |