Congenital HyperinsulinismPhase 2DOPA PETNCT04205604
18FluoroLDOPA PET Imaging for the Detection and Localization of Focal Congenital Hyperinsulinism
A Phase 2 study testing 18F-Fluoro Dopa Imaging in people with Congenital Hyperinsulinism, run by Miguel Pampaloni at 1 site in United States.
Trial summary
The goal of this project is to determine the role of FDOPA/PET as a pre-operative diagnostic imaging procedure for differentiating focal and diffuse forms of congenital hyperinsulinism and locating focal lesions in the pancreas to guide surgical resection. Read more about this study
- Who is it for?
- Ages 0 days to 18 years
- Conditions the study is for
- Congenital Hyperinsulinism
- What is being tested?
- 18F-Fluoro Dopa Imaging
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Nov 2016to 1 Dec 2026
- Phase
- Phase 2
- 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 0 days to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Any age, but primarily infants 0-6 months given typical age of initial presentation.
- Children with diagnosis of FoHI or DiHI based on clinical criteria (fasting hypoglycemia accompanied by inadequate suppression of plasma insulin, inappropriately low plasma free fatty acid and plasma-hydroxybutyrate concentrations, and an inappropriate glycemic response to glucagon injection)
- o confirmed by genetic testing for mutations in ABCC8 and KCNJ1 was1.
- Hypoglycemia uncontrolled with medical management (diazoxide, octreotide).
- Able to withdraw medications in time to wash out prior to the scheduled PET scan.
- Patients fulfilling criteria above but with uncontrolled hypoglycemia after initial surgical management (partial or near-total pancreatectomy)
- Normal hepatic and renal function.
Who cannot join
- Treatment with other, third-line, medications for hyperinsulinism (nifedipine, glucagon).
- Patients with hepatic or renal insufficiency.
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
University of California, San Francisco
San Francisco, California, 94143
Miguel Hernandez Pampaloni, MD, PhD, 415-353-1886
About this study
Official title: 18F-Fluoro-L- DOPA PET Imaging for the Detection and Localization of Focal Congenital Hyperinsulinism
The goal of this project is to determine the role of FDOPA/PET as a pre-operative diagnostic imaging procedure for differentiating focal and diffuse forms of congenital hyperinsulinism and locating focal lesions in the pancreas to guide surgical resection.
Read the detailed description
Congenital hyperinsulinism (HI) is the most common cause of recurrent and persistent hypoglycemia, presenting early in infancy. Patients who fail medical therapy usually require resection of the diseased pancreas(partial or subtotal pancreatectomy) to control this disorder. Over half of patients undergoing surgery have a focal area of islet cell dysfunction that is curable with resection. These focal lesions are areas of adenomatosis consisting of a clone of beta-cells that express a paternally-derived mutation of the KATP channel due to loss of heterozygosity for the maternal allele. Current imaging techniques cannot differentiate focal and diffuse forms of hyperinsulinism, nor can they locate focal areas of disease within the pancreas before surgery. L-DOPA is taken up by some neuroendocrine cells, including pancreatic islet cells, and stored as dopamine in secretory granules. Recent studies show that positron emission tomography (PET) following administration of 18F-fluoro-L-DOPA (FDOPA) can distinguish focal and diffuse forms of HI and accurately locate focal lesions within the pancreas.
- Conditions
- Congenital Hyperinsulinism
- Being tested
- 18F-Fluoro Dopa Imaging
- Medical subject headings
- Congenital Hyperinsulinism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
18F-Fluoro Dopa ImagingDrug
Imaging with 18F-Fluoro Dopa PET Imaging
| Study group | Role | What they receive |
|---|---|---|
| Single Arm, | Experimental | Patients with clinically diagnosed congenital hyperinsulinism Receives: Drug: 18F-Fluoro Dopa Imaging |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sensitivity of Imaging
To determine the sensitivity and specificity of FDOPA PET/CT in differentiating focal and diffuse forms of HI in infants with medically refractory hypoglycemia
Measured: four weeks
Accuracy of Imaging
To determine the accuracy of FDOPA PET/CT localizing areas of islet cell adenomatosis within the pancreas in patients with focal HI, by identifying the area of abnormal uptake of the raioligand
Measured: four weeks
Secondary outcomes (1)
Semiquantitative Imaging Assessment
- determine whether data from FDOPA PET/CT can accurately quantify pancreatic islet cell mass in patients with HI (normal pancreas, areas of adenomatosis in focal HI and diseased pancreas in diffuse HI) by quantifying the standardized uptake value (SUV) of FDOPA of and correlating with histologic findings.
Measured: four weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2019
- Study start3 Nov 2016
- Primary completion1 Dec 2026 (estimated)
- Study completion1 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 2
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Miguel Pampaloni
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Miguel Pampaloni, University of California, San Francisco
- Investigators
- Miguel Hernandez Pampaloni, MD, PhD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04205604
- Sponsor's protocol ID
- 8F-Fluoro-L- DOPA
- Acronym
- DOPA PET
- Status last verified
- Apr 2026
- First submitted
- 16 Dec 2019
- Last updated
- 1 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 18F-Fluoro-L- DOPA PET Imaging for the Detection and Localization of Focal Congenital Hyperinsulinism
- ClinicalTrials.gov ID
- NCT04205604
- Acronym / sponsor ID
- DOPA PET / 8F-Fluoro-L- DOPA
- Conditions
- Congenital Hyperinsulinism
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Miguel PampaloniUniversity, hospital or other
- Study start
- 3 Nov 2016 (actual)
- Primary completion
- 1 Dec 2026 (estimated)
- Study completion
- 1 Dec 2026 (estimated)
- First posted
- 19 Dec 2019 (actual)
- 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.
The goal of this project is to determine the role of FDOPA/PET as a pre-operative diagnostic imaging procedure for differentiating focal and diffuse forms of congenital hyperinsulinism and locating focal lesions in the pancreas to guide surgical resection.
Detailed description
Congenital hyperinsulinism (HI) is the most common cause of recurrent and persistent hypoglycemia, presenting early in infancy. Patients who fail medical therapy usually require resection of the diseased pancreas(partial or subtotal pancreatectomy) to control this disorder. Over half of patients undergoing surgery have a focal area of islet cell dysfunction that is curable with resection. These focal lesions are areas of adenomatosis consisting of a clone of beta-cells that express a paternally-derived mutation of the KATP channel due to loss of heterozygosity for the maternal allele. Current imaging techniques cannot differentiate focal and diffuse forms of hyperinsulinism, nor can they locate focal areas of disease within the pancreas before surgery. L-DOPA is taken up by some neuroendocrine cells, including pancreatic islet cells, and stored as dopamine in secretory granules. Recent studies show that positron emission tomography (PET) following administration of 18F-fluoro-L-DOPA (FDOPA) can distinguish focal and diffuse forms of HI and accurately locate focal lesions within the pancreas.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm, | Experimental | Drug: 18F-Fluoro Dopa ImagingPatients with clinically diagnosed congenital hyperinsulinism |
Who may qualify
- Ages
- 0 days to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Any age, but primarily infants 0-6 months given typical age of initial presentation.
- Children with diagnosis of FoHI or DiHI based on clinical criteria (fasting hypoglycemia accompanied by inadequate suppression of plasma insulin, inappropriately low plasma free fatty acid and plasma-hydroxybutyrate concentrations, and an inappropriate glycemic response to glucagon injection)
- o confirmed by genetic testing for mutations in ABCC8 and KCNJ1 was1.
- Hypoglycemia uncontrolled with medical management (diazoxide, octreotide).
- Able to withdraw medications in time to wash out prior to the scheduled PET scan.
Key exclusion criteria (2 listed)
- Treatment with other, third-line, medications for hyperinsulinism (nifedipine, glucagon).
- Patients with hepatic or renal insufficiency.
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 |
|---|---|---|
| 18F-Fluoro Dopa ImagingImaging with 18F-Fluoro Dopa PET Imaging | Drug | Single Arm, |
Primary outcome
Sensitivity of Imaging
Time frame: four weeks
To determine the sensitivity and specificity of FDOPA PET/CT in differentiating focal and diffuse forms of HI in infants with medically refractory hypoglycemia
Accuracy of Imaging
Time frame: four weeks
To determine the accuracy of FDOPA PET/CT localizing areas of islet cell adenomatosis within the pancreas in patients with focal HI, by identifying the area of abnormal uptake of the raioligand
1 secondary outcome
Semiquantitative Imaging Assessment
Time frame: four weeks
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 1 May 2026.
- Individual participant dataSharing planned: No
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.
Miguel Hernandez Pampaloni, MD, PhD principal investigator
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 18F-Fluoro Dopa Imaging in people with Congenital Hyperinsulinism, run by Miguel Pampaloni 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 7 inclusion and 2 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 1 May 2026. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Sensitivity of Imaging (time frame: four weeks). 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?
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 1 May 2026.