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| ID | Type | Description | Link |
|---|---|---|---|
| FD-R-003457-01 | Other Grant/Funding Number | FDA orphan drug grant |
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| Name | Class |
|---|---|
| University of Pennsylvania | OTHER |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Subjects who had PET and surgery | Experimental | Children diagnosed with hyperinsulinism who have failed other non-surgical interventions and are candidates to be scheduled for surgery for partial pancreatectomy. Eligible children will undergo PET imaging with F-DOPA prior to surgery. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| F-DOPA | Drug | 0.08-0.16 mCi/kg once |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 | up to 1 month post surgical intervention |
| Measure | Description | Time Frame |
|---|---|---|
| Safety of F-DOPA PET | Number of Participants with Adverse Events | 72 hours maximum or prior to pancreatic surgery |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Lisa J States, MD | Children's Hospital of Philadelphia | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 17564642 | Background | Hardy OT, Litman RS. Congenital hyperinsulinism - a review of the disorder and a discussion of the anesthesia management. Paediatr Anaesth. 2007 Jul;17(7):616-21. doi: 10.1111/j.1460-9592.2007.02192.x. | |
| 16380471 | Background | Otonkoski T, Nanto-Salonen K, Seppanen M, Veijola R, Huopio H, Hussain K, Tapanainen P, Eskola O, Parkkola R, Ekstrom K, Guiot Y, Rahier J, Laakso M, Rintala R, Nuutila P, Minn H. Noninvasive diagnosis of focal hyperinsulinism of infancy with [18F]-DOPA positron emission tomography. Diabetes. 2006 Jan;55(1):13-8. |
| Label | URL |
|---|---|
| The Congenital Hyperinsulinism Center at The Children's Hospital of Philadelphia | View source |
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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.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | All Subjects Consented | There were 106 subjects consented to participate. 105 actually underwent PET scan. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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| ID | Title | Description |
|---|---|---|
| BG000 | Subjects Who Consented | These demographics include all subject recruited who consented whether they are evaluable or not |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 | 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). | Posted | Count of Participants | Participants | up to 1 month post surgical intervention |
|
Reviewed within 72 hours post PET or prior to pancreatic surgery (which ever was first).
Included all participants who had PET(whether or not they also had surgery).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | All Subjects Who Had PET | 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 18 F-DOPA: 1 time injection of 0.08 - 0.16 mCi/kg of 18F-DOPA |
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Some subjects have atypical disease that is neither focal or diffuse. Rapid genetic test results were not available at the beginning of the study.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Lisa J. States, MD | Children's Hospital of Philadelphia/Perelman School of Medicine, University of Pennsylvania | 267-425-7146 | states@chop.edu |
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| ID | Term |
|---|---|
| D044903 | Congenital Hyperinsulinism |
| D006946 | Hyperinsulinism |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D007232 | Infant, Newborn, Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C043437 | fluorodopa F 18 |
| D009682 | Magnetic Resonance Spectroscopy |
| ID | Term |
|---|---|
| D013057 | Spectrum Analysis |
| D002623 | Chemistry Techniques, Analytical |
| D008919 | Investigative Techniques |
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| PET scan | Radiation |
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| 17923769 | Background | Ribeiro MJ, Boddaert N, Delzescaux T, Valayannopoulos V, Bellanne-Chantelot C, Jaubert F, Verkarre V, Nihoul-Fekete C, Brunelle F, Lonlay P. Functional imaging of the pancreas: the role of [18F]fluoro-L-DOPA PET in the diagnosis of hyperinsulinism of infancy. Endocr Dev. 2007;12:55-66. doi: 10.1159/000109605. |
| 17895790 | Background | Subramaniam RM, Karantanis D, Peller PJ. [18F]Fluoro-L-dopa PET/CT in congenital hyperinsulinism. J Comput Assist Tomogr. 2007 Sep-Oct;31(5):770-2. doi: 10.1097/RCT.0b013e318031f55c. |
| 17661030 | Background | Ribeiro MJ, Boddaert N, Bellanne-Chantelot C, Bourgeois S, Valayannopoulos V, Delzescaux T, Jaubert F, Nihoul-Fekete C, Brunelle F, De Lonlay P. The added value of [18F]fluoro-L-DOPA PET in the diagnosis of hyperinsulinism of infancy: a retrospective study involving 49 children. Eur J Nucl Med Mol Imaging. 2007 Dec;34(12):2120-8. doi: 10.1007/s00259-007-0498-y. Epub 2007 Jul 28. |
| 16403819 | Background | de Lonlay P, Simon-Carre A, Ribeiro MJ, Boddaert N, Giurgea I, Laborde K, Bellanne-Chantelot C, Verkarre V, Polak M, Rahier J, Syrota A, Seidenwurm D, Nihoul-Fekete C, Robert JJ, Brunelle F, Jaubert F. Congenital hyperinsulinism: pancreatic [18F]fluoro-L-dihydroxyphenylalanine (DOPA) positron emission tomography and immunohistochemistry study of DOPA decarboxylase and insulin secretion. J Clin Endocrinol Metab. 2006 Mar;91(3):933-40. doi: 10.1210/jc.2005-1713. Epub 2006 Jan 10. |
| 17895314 | Result | Hardy OT, Hernandez-Pampaloni M, Saffer JR, Scheuermann JS, Ernst LM, Freifelder R, Zhuang H, MacMullen C, Becker S, Adzick NS, Divgi C, Alavi A, Stanley CA. Accuracy of [18F]fluorodopa positron emission tomography for diagnosing and localizing focal congenital hyperinsulinism. J Clin Endocrinol Metab. 2007 Dec;92(12):4706-11. doi: 10.1210/jc.2007-1637. Epub 2007 Sep 25. |
| 17236890 | Result | Hardy OT, Hernandez-Pampaloni M, Saffer JR, Suchi M, Ruchelli E, Zhuang H, Ganguly A, Freifelder R, Adzick NS, Alavi A, Stanley CA. Diagnosis and localization of focal congenital hyperinsulinism by 18F-fluorodopa PET scan. J Pediatr. 2007 Feb;150(2):140-5. doi: 10.1016/j.jpeds.2006.08.028. |
| International non-profit organization created by parents/families based in the United States | View source |
| Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Count of Participants | Participants |
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| Secondary | Safety of F-DOPA PET | Number of Participants with Adverse Events | Posted | Count of Participants | Participants | 72 hours maximum or prior to pancreatic surgery |
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| 0 |
| 105 |
| 0 |
| 105 |
| 0 |
| 105 |
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| D044882 | Glucose Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D007003 | Hypoglycemia |