Congenital HyperinsulinismPhase 1NCT02021604
Fluorodopa F 18 in Congenital Hyperinsulinism and Insulinoma
A Phase 1 study testing Fluorodopa F 18 in people with Congenital Hyperinsulinism and Insulinoma, run by Cook Children's Health Care System at 1 site in United States.
Trial summary
Low blood sugars are known to cause brain damage in newborn babies. One of the most common causes of low blood sugars persisting beyond the new born period is a condition called congenital hyperinsulinism (HI). This is a disease whereby the pancreas secretes too much insulin and causes low blood sugars. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Congenital Hyperinsulinism, Insulinoma
- What is being tested?
- Fluorodopa F 18
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 250 participants
- Where
- 1 siteUnited States
- Study dates
- 9 Oct 2013to Jun 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with HI attending the Cook Children's Congenital Hyperinsulinism Center and being treated by an Endocrinologist which may be the PI or a partner of this clinician.
- The patient's Endocrinologist has determined that the patient cannot be safely managed with standard medical therapy (failed) and surgery is recommended to prevent future episodes of severe hypoglycemia and preserve brain function. Failure of medical therapy is defined as both:
- Hypoglycemia (blood glucose <70 m/dL) on a single measure despite the use of anti-hypoglycemic medications, if applicable to the individual patient, including and limited to diazoxide or octreotide
- Inability to fast, defined as the inability to maintain a blood glucose >50 mg/dL for: 1) more than 12 hours for infants < 1 year of age; 2) more than 15 hours 1-3 years of age; 3) more than 18 hours over 3 years of age
- Patients in whom the genetic testing (if available and informative) does not prove diffuse HI disease. Such children might be considered if they have one or more of the following situations:
- no genetic testing results (e.g., due to insurance denial or parental refusal)
Show all 10Show fewer
- negative genetic testing (note: only 75% of mutations may be found with existing technology)
- no autosomal recessive mutations in ABCC8 or KCNJ11 on the maternal allele
- no autosomal dominant mutations in ABCC8 or KCNJ11
- Patients thought to have focal HI disease based on genetic testing or insulinoma based on clinical evaluation and have well-controlled blood glucose levels with any degree of dietary or medical management, BUT the patient and their parent(s) or LAR wishes to proceed with surgery for a possible cure of HI disease.
Who cannot join
- Patients who do not have a diagnosis of HI
- Patients with genetic evidence of diffuse HI
- Patients who are pregnant
- Nursing mothers who are unwilling to discontinue breastfeeding their infant for 48 hours after Fluorodopa F 18 injection
- Patients with a known allergy to Fluorodopa F 18 agent
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Cook Children's Medical Center
Fort Worth, Texas, 76104
Paul Thornton, MD
About this study
Official title: The Use of Fluorodopa F 18 Positron Emission Tomography Combined With Computed Tomography in Congenital Hyperinsulinism and Insulinoma
Low blood sugars are known to cause brain damage in newborn babies. One of the most common causes of low blood sugars persisting beyond the new born period is a condition called congenital hyperinsulinism (HI). This is a disease whereby the pancreas secretes too much insulin and causes low blood sugars. Twenty to forty percent of these babies will have brain damage. There are two forms of this disease. In one form only a small part of the pancreas makes too much insulin (focal HI) and in the other, the whole pancreas make too much insulin (diffuse HI). Another very similar disease is insulinoma which occurs after birth, but also causes hyperinsulinism. If a surgeon could know which part of the pancreas has the focal lesion he could remove it and cure the patient.
The purpose of this study is to investigate whether a new investigational drug called Fluorodopa F 18, when used with a PET scan, can find the focal lesion and guide the surgeon to remove it, thus curing the patient and preventing further brain damage.
Read the detailed description
Congenital Hyperinsulinism (HI) is a disorder of insulin secretion that causes profound hypoglycemia leading to significant morbidity. It is the most common form of persistent neonatal hypoglycemia, and the most dangerous. Inappropriate insulin secretion causes not only hypoglycemia but also inability to release free fatty acids from adipose tissue and inability to release glycogen from the liver. Hence the brain is deprived of all the major fuel sources (glucose, beta-hydroxybutyrate and lactate) for energy thus leading to the high incidence of brain damage. In up to 50% of cases of congenital hyperinsulinism, medical therapy fails and surgical resection of the pancreas is necessary. Previous techniques developed to differentiate those patients with focal HI in whom surgery could result in a cure are very invasive and put the infants at risk for hypoglycemic brain damage or arterial thrombosis.
It is known that the beta cells in the pancreatic islets, similar to other neuroendocrine tissues, contain amino acid decarboxylase (AADC). Beta cells take up L-Dopa and convert it into dopamine by AADC. It was proposed that as other neuroendocrine tumors such as phaeochromocytoma and carcinoid tumors express AADC and can be very easily visualized using Fluorodopa F 18 PET then so also would the pancreas be easily and accurately visualized. After initial reports demonstrated the effectiveness, safety and accuracy, there have been now over 200 patients with HI reported in the literature who have had Fluorodopa F 18 PET scans with suggestions that referral to major HI centers for Fluorodopa F 18 PET CT is now an integral part of standard of care management of patients with HI that require surgery. Fluorodopa F 18 PET scanning for patients with Hyperinsulinism is now established in Europe and Australia, and has close to 95% sensitivity. When linked to Computed Tomography (CT) image of the pancreas, Fluorodopa F 18 PET allows the surgeon to image the pancreas in three dimensions, to even more accurately identify the site of the focal lesion, increasing the chance of a sufficient partial pancreatectomy to cure the patient.
Similarly, insulinomas are neuroendocrine cell tumors that are typically benign (90%) and very rare occurring in 1-4 per million of the population with > 50% occurring in adults >age 25 years. Biochemical differentiation of insulinomas from congenital hyperinsulinism may be aided by the use of the pro-insulin:insulin ratio, by the age of presentation and by the history. The standard of care for insulinoma is to remove them once identified, rather than long term medical treatment. Current imaging techniques include CT scan pancreas, endoscopic ultrasound of the pancreas and MRI pancreas however despite using these modalities in some patients the insulinoma may not be found. 18F-DOPA has been shown to be superior than MIBG scanning for neuroendocrine tumors such as phaeochromocytoma, but there is very little data in patients with insulinoma. Moreover, patients with MEN 1 and insulinoma may have more than one tumor, which if missed with conventional imaging could result in failure to cure with surgery.
The objectives of this study are:
- To determine, using Positron Emission Tomography, whether or not the uptake of a radiopharmaceutical agent, Fluorodopa 18F (18F-DOPA) produced in a cyclotron located at a distance far from the imaging center will produce qualitatively adequate pancreatic images in patients with congenital hyperinsulinism
- To determine, using direct comparisons, whether or not Fluorodopa 18F Positron Emission Tomography (18F-DOPA PET) combined with Computed Tomography (CT) will produce pancreatic images matching the gold-standard of histopathological findings at surgery for partial or complete pancreatectomy in the treatment of patients with congenital hyperinsulinism
- To determine, using direct comparisons, whether or not Fluorodopa 18F Positron Emission Tomography (18F-DOPA PET) combined with Computed Tomography (CT) will produce pancreatic images matching the gold-standard of histopathological findings at surgery for insulinomas
- To determine the best way to interpret the 18F-DOPA PET scans comparing SUV max:SUV sub max at a ratio of the current 1.5, a suggested 1.3 and by using visual inspection of the images.
- Conditions
- Congenital Hyperinsulinism, Insulinoma
- Being tested
- Fluorodopa F 18
- Keywords
- Congenital Hyperinsulinism, HI, Hypoglycemia, FDOPA, 18F-DOPA, Hyperinsulinism, Insulinoma
- Medical subject headings
- Congenital Hyperinsulinism, Insulinoma, Hypoglycemia, Hyperinsulinism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fluorodopa F 18Drug
Also called Fluorodopa 18F (International Non-Proprietary Name), 6-[18F]fluorolevodopa (U.S. Pharmacopeia), Fluorodopa F 18 (CAS Number 75290-51-6)
A dose of Fluorodopa F 18, 3-6 MBq/Kg (0.08-0.16 mCi/kg), will be injected intravenously into the subject under the direct supervision of the radiology sub-investigator. Then, the PET imaging procedure will begin and proceed for up to 70 minutes after injection. An abdominal CT image will be made using intravenous contrast. Both images, PET and CT, will be co-localized by the radiologist for interpretation.
| Study group | Role | What they receive |
|---|---|---|
| Pancreatic Imaging with Fluorodopa F 18 | Experimental | Not described Receives: Drug: Fluorodopa F 18 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Radioactivity of 18F-DOPA following transport
Positron Emission Tomography will be used to determine whether or not the uptake of a radiopharmaceutical agent, Fluorodopa F 18, produced in a cyclotron located at a distance far from the imaging center will produce qualitatively adequate pancreatic images in patients with congenital hyperinsulinism
Measured: 1 day
Accuracy of PET imaging compared to intraoperative pancreatic biopsy in patients with congenital hyperinsulinism
Investigators will directly compare pancreatic images from Fluorodopa F 18 PET combined with Computed Tomography versus the gold-standard of histopathological findings at surgery in subjects who received a partial or complete pancreatectomy
Measured: up to one month
Secondary outcomes (2)
Accuracy of PET imaging compared to intraoperative pancreatic biopsy in patients with insulinoma
Investigators will directly compare pancreatic images from Fluorodopa F 18 PET combined with Computed Tomography versus the gold-standard of histopathological findings at surgery in subjects who received a partial or complete pancreatectomy
Measured: up to one month
Ratio of Standard Uptake Value max to sub max
Investigators will compare SUV max to SUV sub max at a ratio of the current 1.5, a suggested 1.3 and by using visual inspection of the images.
Measured: up to one month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Dec 2013 (estimated)
- Study start9 Oct 2013
- Primary completionJan 2028 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for Congenital HyperinsulinismStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 250 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cook Children's Health Care System
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Paul Thornton, MD, Principal investigator, Cook Children's Health Care System
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02021604
- Sponsor's protocol ID
- 2012-060
- Status last verified
- Jul 2024
- First submitted
- 20 Dec 2013
- Last updated
- 16 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
de Lonlay-Debeney P, Poggi-Travert F, Fournet JC, Sempoux C, Dionisi Vici C, Brunelle F, Touati G, Rahier J, Junien C, Nihoul-Fekete C, Robert JJ, Saudubray JM. Clinical features of 52 neonates with hyperinsulinism. N Engl J Med. 1999 Apr 15;340(15):1169-75. doi: 10.1056/NEJM199904153401505.
PubMed 10202168Thornton PS, Finegold DN, Stanley CA, Sperling MA. Hypoglycemia in the infant and child. In Sperling MA ed. Pediatric Endocrinology 2nd ed., pp 367-84. Philadelphia: Saunders, 2002.
Stanley CA, Thornton PS, Finegold DN, Sperling MA: Hypoglycemia in neonates and infants. In Sperling MA ed. Pediatric Endocrinology 2nd edition chpt 7 pages 135-59. 2002.
Suchi M, Thornton PS, Adzick NS, MacMullen C, Ganguly A, Stanley CA, Ruchelli ED. Congenital hyperinsulinism: intraoperative biopsy interpretation can direct the extent of pancreatectomy. Am J Surg Pathol. 2004 Oct;28(10):1326-35. doi: 10.1097/01.pas.0000138000.61897.32.
PubMed 15371948Jager PL, Chirakal R, Marriott CJ, Brouwers AH, Koopmans KP, Gulenchyn KY. 6-L-18F-fluorodihydroxyphenylalanine PET in neuroendocrine tumors: basic aspects and emerging clinical applications. J Nucl Med. 2008 Apr;49(4):573-86. doi: 10.2967/jnumed.107.045708. Epub 2008 Mar 14.
PubMed 18344441Otonkoski 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.
PubMed 16380471Mohnike K, Blankenstein O, Minn H, Mohnike W, Fuchtner F, Otonkoski T. [18F]-DOPA positron emission tomography for preoperative localization in congenital hyperinsulinism. Horm Res. 2008;70(2):65-72. doi: 10.1159/000137655. Epub 2008 Jun 12.
PubMed 18547951Hardy 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.
PubMed 17236890Hardy 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.
PubMed 17895314
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Use of Fluorodopa F 18 Positron Emission Tomography Combined With Computed Tomography in Congenital Hyperinsulinism and Insulinoma
- ClinicalTrials.gov ID
- NCT02021604
- Acronym / sponsor ID
- 2012-060
- Conditions
- Congenital Hyperinsulinism, Insulinoma
- Keywords
- Congenital Hyperinsulinism, HI, Hypoglycemia, FDOPA, 18F-DOPA, Hyperinsulinism, Insulinoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- Cook Children's Health Care SystemUniversity, hospital or other
- Study start
- 9 Oct 2013 (actual)
- Primary completion
- Jan 2028 (estimated)
- Study completion
- Jun 2028 (estimated)
- First posted
- 27 Dec 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Low blood sugars are known to cause brain damage in newborn babies. One of the most common causes of low blood sugars persisting beyond the new born period is a condition called congenital hyperinsulinism (HI). This is a disease whereby the pancreas secretes too much insulin and causes low blood sugars. Twenty to forty percent of these babies will have brain damage. There are two forms of this disease. In one form only a small part of the pancreas makes too much insulin (focal HI) and in the other, the whole pancreas make too much insulin (diffuse HI). Another very similar disease is insulinoma which occurs after birth, but also causes hyperinsulinism. If a surgeon could know which part of the pancreas has the focal lesion he could remove it and cure the patient.
The purpose of this study is to investigate whether a new investigational drug called Fluorodopa F 18, when used with a PET scan, can find the focal lesion and guide the surgeon to remove it, thus curing the patient and preventing further brain damage.
Detailed description
Congenital Hyperinsulinism (HI) is a disorder of insulin secretion that causes profound hypoglycemia leading to significant morbidity. It is the most common form of persistent neonatal hypoglycemia, and the most dangerous. Inappropriate insulin secretion causes not only hypoglycemia but also inability to release free fatty acids from adipose tissue and inability to release glycogen from the liver. Hence the brain is deprived of all the major fuel sources (glucose, beta-hydroxybutyrate and lactate) for energy thus leading to the high incidence of brain damage. In up to 50% of cases of congenital hyperinsulinism, medical therapy fails and surgical resection of the pancreas is necessary. Previous techniques developed to differentiate those patients with focal HI in whom surgery could result in a cure are very invasive and put the infants at risk for hypoglycemic brain damage or arterial thrombosis.
It is known that the beta cells in the pancreatic islets, similar to other neuroendocrine tissues, contain amino acid decarboxylase (AADC). Beta cells take up L-Dopa and convert it into dopamine by AADC. It was proposed that as other neuroendocrine tumors such as phaeochromocytoma and carcinoid tumors express AADC and can be very easily visualized using Fluorodopa F 18 PET then so also would the pancreas be easily and accurately visualized. After initial reports demonstrated the effectiveness, safety and accuracy, there have been now over 200 patients with HI reported in the literature who have had Fluorodopa F 18 PET scans with suggestions that referral to major HI centers for Fluorodopa F 18 PET CT is now an integral part of standard of care management of patients with HI that require surgery. Fluorodopa F 18 PET scanning for patients with Hyperinsulinism is now established in Europe and Australia, and has close to 95% sensitivity. When linked to Computed Tomography (CT) image of the pancreas, Fluorodopa F 18 PET allows the surgeon to image the pancreas in three dimensions, to even more accurately identify the site of the focal lesion, increasing the chance of a sufficient partial pancreatectomy to cure the patient.
Similarly, insulinomas are neuroendocrine cell tumors that are typically benign (90%) and very rare occurring in 1-4 per million of the population with > 50% occurring in adults >age 25 years. Biochemical differentiation of insulinomas from congenital hyperinsulinism may be aided by the use of the pro-insulin:insulin ratio, by the age of presentation and by the history. The standard of care for insulinoma is to remove them once identified, rather than long term medical treatment. Current imaging techniques include CT scan pancreas, endoscopic ultrasound of the pancreas and MRI pancreas however despite using these modalities in some patients the insulinoma may not be found. 18F-DOPA has been shown to be superior than MIBG scanning for neuroendocrine tumors such as phaeochromocytoma, but there is very little data in patients with insulinoma. Moreover, patients with MEN 1 and insulinoma may have more than one tumor, which if missed with conventional imaging could result in failure to cure with surgery.
The objectives of this study are:
- To determine, using Positron Emission Tomography, whether or not the uptake of a radiopharmaceutical agent, Fluorodopa 18F (18F-DOPA) produced in a cyclotron located at a distance far from the imaging center will produce qualitatively adequate pancreatic images in patients with congenital hyperinsulinism
- To determine, using direct comparisons, whether or not Fluorodopa 18F Positron Emission Tomography (18F-DOPA PET) combined with Computed Tomography (CT) will produce pancreatic images matching the gold-standard of histopathological findings at surgery for partial or complete pancreatectomy in the treatment of patients with congenital hyperinsulinism
- To determine, using direct comparisons, whether or not Fluorodopa 18F Positron Emission Tomography (18F-DOPA PET) combined with Computed Tomography (CT) will produce pancreatic images matching the gold-standard of histopathological findings at surgery for insulinomas
- To determine the best way to interpret the 18F-DOPA PET scans comparing SUV max:SUV sub max at a ratio of the current 1.5, a suggested 1.3 and by using visual inspection of the images.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pancreatic Imaging with Fluorodopa F 18 | Experimental | Drug: Fluorodopa F 18 |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Patients with HI attending the Cook Children's Congenital Hyperinsulinism Center and being treated by an Endocrinologist which may be the PI or a partner of this clinician.
- The patient's Endocrinologist has determined that the patient cannot be safely managed with standard medical therapy (failed) and surgery is recommended to prevent future episodes of severe hypoglycemia and preserve brain function. Failure of medical therapy is defined as both:
- Patients in whom the genetic testing (if available and informative) does not prove diffuse HI disease. Such children might be considered if they have one or more of the following situations:
- Patients thought to have focal HI disease based on genetic testing or insulinoma based on clinical evaluation and have well-controlled blood glucose levels with any degree of dietary or medical management, BUT the patient and their parent(s) or LAR wishes to proceed with surgery for a possible cure of HI disease.
Key exclusion criteria (5 listed)
- Patients who do not have a diagnosis of HI
- Patients with genetic evidence of diffuse HI
- Patients who are pregnant
- Nursing mothers who are unwilling to discontinue breastfeeding their infant for 48 hours after Fluorodopa F 18 injection
- Patients with a known allergy to Fluorodopa F 18 agent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fluorodopa F 18Also: Fluorodopa 18F (International Non-Proprietary Name), 6-[18F]fluorolevodopa (U.S. Pharmacopeia), Fluorodopa F 18 (CAS Number 75290-51-6)A dose of Fluorodopa F 18, 3-6 MBq/Kg (0.08-0.16 mCi/kg), will be injected intravenously into the subject under the direct supervision of the radiology sub-investigator. Then, the PET imaging procedure will begin and proceed for up to 70 minutes after injection. An abdominal CT image will be made using intravenous contrast. Both images, PET and CT, will be co-localized by the radiologist for interpretation. | Drug | Pancreatic Imaging with Fluorodopa F 18 |
Primary outcome
Radioactivity of 18F-DOPA following transport
Time frame: 1 day
Positron Emission Tomography will be used to determine whether or not the uptake of a radiopharmaceutical agent, Fluorodopa F 18, produced in a cyclotron located at a distance far from the imaging center will produce qualitatively adequate pancreatic images in patients with congenital hyperinsulinism
Accuracy of PET imaging compared to intraoperative pancreatic biopsy in patients with congenital hyperinsulinism
Time frame: up to one month
Investigators will directly compare pancreatic images from Fluorodopa F 18 PET combined with Computed Tomography versus the gold-standard of histopathological findings at surgery in subjects who received a partial or complete pancreatectomy
2 secondary outcomes
Accuracy of PET imaging compared to intraoperative pancreatic biopsy in patients with insulinoma
Time frame: up to one month
Ratio of Standard Uptake Value max to sub max
Time frame: up to one month
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2024.
- Background: PubMed 10202168de Lonlay-Debeney P, Poggi-Travert F, Fournet JC, Sempoux C, Dionisi Vici C, Brunelle F, Touati G, Rahier J, Junien C, Nihoul-Fekete C, Robert JJ, Saudubray JM. Clinical features of 52 neonates with hyperinsulinism. N Engl J Med. 1999 Apr 15;340(15):1169-75. doi: 10.1056/NEJM199904153401505.
- BackgroundThornton PS, Finegold DN, Stanley CA, Sperling MA. Hypoglycemia in the infant and child. In Sperling MA ed. Pediatric Endocrinology 2nd ed., pp 367-84. Philadelphia: Saunders, 2002.
- BackgroundStanley CA, Thornton PS, Finegold DN, Sperling MA: Hypoglycemia in neonates and infants. In Sperling MA ed. Pediatric Endocrinology 2nd edition chpt 7 pages 135-59. 2002.
- Background: PubMed 15371948Suchi M, Thornton PS, Adzick NS, MacMullen C, Ganguly A, Stanley CA, Ruchelli ED. Congenital hyperinsulinism: intraoperative biopsy interpretation can direct the extent of pancreatectomy. Am J Surg Pathol. 2004 Oct;28(10):1326-35. doi: 10.1097/01.pas.0000138000.61897.32.
- Background: PubMed 18344441Jager PL, Chirakal R, Marriott CJ, Brouwers AH, Koopmans KP, Gulenchyn KY. 6-L-18F-fluorodihydroxyphenylalanine PET in neuroendocrine tumors: basic aspects and emerging clinical applications. J Nucl Med. 2008 Apr;49(4):573-86. doi: 10.2967/jnumed.107.045708. Epub 2008 Mar 14.
- Background: PubMed 16380471Otonkoski 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.
- Background: PubMed 18547951Mohnike K, Blankenstein O, Minn H, Mohnike W, Fuchtner F, Otonkoski T. [18F]-DOPA positron emission tomography for preoperative localization in congenital hyperinsulinism. Horm Res. 2008;70(2):65-72. doi: 10.1159/000137655. Epub 2008 Jun 12.
- Background: PubMed 17236890Hardy 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.
- Background: PubMed 17895314Hardy 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.
Related trials for Congenital Hyperinsulinism
RecruitingObservational study
Asprosin and miRNA Expression in Young Women With Metabolic Syndrome
Metabolic Syndrome, Insulin Resistance
RecruitingObservational study
Metabolic Phenotyping in vEDS
Vascular Ehlers-Danlos Syndrome, Vascular Ehlers Danlos Syndrome
Recruiting
Metabolic Syndrome Resposne to Dietary Limitations in Familial Mediterranean Fever in Adolescents
Familial Mediterranean Fever (FMF ), Metabolic Syndrome
Not yet recruitingPhase 4
Mazdutide for Weight Management and Gout in Adults With Obesity
Gout, Obesity (BMI>30)
RecruitingObservational study
Polycystic Ovary Syndrome in Type 1 Diabetes
Type 1 Diabetes Mellitus, Polycystic Ovary Syndrome (PCOS)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Paul Thornton, MD principal investigator
Cook Children's Health Care System
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Fluorodopa F 18 in people with Congenital Hyperinsulinism and Insulinoma, run by Cook Children's Health Care System at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Deborah Rafferty, PhD (682-303-1363, Deborah.Rafferty@cookchildrens.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Radioactivity of 18F-DOPA following transport (time frame: 1 day). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02021604, which also lists 9 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2024.