Phase II Safety and Efficacy Study of 18FDOPA PET-CT in Children With Hyperinsulinemic Hypoglycemia

Study Overview
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Brief Summary
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Children with congenital hyperinsulinism (CHI) have low blood sugar, and some of these children may require surgery to remove part or all of their pancreas. In this study, researchers will test how well a radioactive drug, 18-labeled L-fluorodeoxyphenylalanine (called F-DOPA) can detect a form of hyperinsulinism (focal HI) that may be cured by surgery. Eligible participants in this study will have positron emission tomography/computerized tomography (PET/CT) scans with F-DOPA prior to surgery.

Detailed Description
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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 (called diffuse disease); others have them located in specific areas of the pancreas (called focal disease). Children who have focal disease located in specific areas of the pancreas may be cured with surgery. F-DOPA is a radioactive drug that is picked up by these cells and used for positron emission tomography (or PET), an imaging technique used in nuclear medicine departments. In this study, researchers will validate the efficacy and safety of using PET/CT with F-DOPA in the pre-operative localization of focal disease in children with hyperinsulinism.

Official Title
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A Phase II Safety and Efficacy Study of 18F-L-Fluoro-DOPA PET/CT Scan Localization of Focal Pancreatic Lesions in Children With Hyperinsulinemic Hypoglycemia

Conditions
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Congenital Hyperinsulinism (CHI), Persistent Hyperinsulinemic Hypoglycemia of Infancy (PHHI)

Intervention / Treatment
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* Drug: 18 F-DOPA


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Infants and children with a clinical diagnosis of hyperinsulinism who are suspected to have focal disease and are surgical candidates for pancreatectomy Exclusion Criteria: Pregnant or lactating females Any other major illness or condition that might substantially increase the risk associated with the subject's participation in this study.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Diagnostic
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: (18F-DOPA) PET/CT imaging<br>Obtain safety and efficacy data on the use of 18-labeled L-fluorodeoxyphenylalanine (18F-DOPA) PET imaging in children with HI for the clinical indication of localizing a focal lesion | Drug: 18 F-DOPA<br>* 1 time injection of 0.08 - 0.16 mCi/kg of 18F-DOPA<br>* Other names: 18FDOPA PET SCAN;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Accuracy of 18F-DOPA PET/CT Scans to Detect Focal Lesions in Children With Congenital Hyperinsulinism | 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. | Surgery typically occured within a week post PET | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety of 18F-DOPA PET/CT Scan - Number of Participants With Adverse Events | 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 | evaluated with 72 hours or prior to pancreatic surgery (if any) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CHI, Congenital Hyperinsulinism (CHI), hyperinsulinism, Persistent Hyperinsulinemic Hypoglycemia of Infancy (PHHI), PHHI, Pancreatectomy, 18F-DOPA, PET/CT scan