Fluorodopa F 18 for Congenital Hyperinsulinism

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Cook Children's Medical Center, Fort Worth, TX
Congenital Hyperinsulinism+3 More
Fluorodopa F 18 - Drug
Eligibility
< 65
All Sexes
Eligible conditions
Select

Study Summary

Fluorodopa F 18 in Congenital Hyperinsulinism and Insulinoma

See full description

Eligible Conditions

  • Congenital Hyperinsulinism
  • Insulinoma

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Fluorodopa F 18 will improve 2 primary outcomes and 2 secondary outcomes in patients with Congenital Hyperinsulinism. Measurement will happen over the course of 1 day.

1 day
Radioactivity of 18F-DOPA following transport
up to one month
Accuracy of PET imaging compared to intraoperative pancreatic biopsy in patients with congenital hyperinsulinism
Accuracy of PET imaging compared to intraoperative pancreatic biopsy in patients with insulinoma
Ratio of Standard Uptake Value max to sub max

Trial Safety

Safety Estimate

1 of 3

Trial Design

1 Treatment Group

Pancreatic Imaging with Fluorodopa F 18
1 of 1
Experimental Treatment

This trial requires 250 total participants across 1 different treatment group

This trial involves a single treatment. Fluorodopa F 18 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.

Pancreatic Imaging with Fluorodopa F 18
Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to one month
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to one month for reporting.

Closest Location

Cook Children's Medical Center - Fort Worth, TX

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 8 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Patients who have HI and are being treated by an endocrinologist, either the PI or a partner, attend the Cook Children's Congenital Hyperinsulinism Center. show original
Some patients with focal HI disease, based on genetic testing or insulinoma based on clinical evaluation, have well-controlled blood glucose levels with any degree of dietary or medical management show original
and/or insulin secretagogues If someone has blood glucose levels that are lower than normal, despite taking medications to help control them, this may be a sign of hypoglycemia show original
) People who are unable to fast are those who cannot maintain a blood glucose level above 50 mg/dL for more than 12 hours for infants under 1 year old, more than 15 hours for children aged 1-3 years, and more than 18 hours for those aged 3 years or older. show original
were found The study found that no autosomal dominant mutations in ABCC8 or KCNJ11 were found. show original
Genetic testing can sometimes identify genetic mutations that increase the risk of developing a particular disease show original
There are no mutations on the maternal allele of ABCC8 or KCNJ11 that are autosomal recessive. show original
were available No genetic testing results were available, due to insurance denial or parental refusal. show original

Patient Q&A Section

What causes congenital hyperinsulinism?

"The pathophysiology of congenital hyperinsulinism is not entirely known. In a recent study, findings, the authors describe one case of congenital hyperinsulinism whose cause remained controversial for several decades." - Anonymous Online Contributor

Unverified Answer

How many people get congenital hyperinsulinism a year in the United States?

"The prevalence of CHI is 1 in 1,500 to 3,000 live births in the United States. With the incidence of CHI per year of approximately 2/100,000 live births, the prevalence of CHI per year in the United States should increase the number of cases being treated by pediatric endocrinology, because many cases of HHI will not be detected." - Anonymous Online Contributor

Unverified Answer

What are common treatments for congenital hyperinsulinism?

"Without specific genetic or medical testing or therapy, most children with HHI will be managed medically with frequent glucose checks, insulin injections, monitoring for hypoglycaemia, and/or lifestyle adjustments. Very few children will require surgery." - Anonymous Online Contributor

Unverified Answer

What are the signs of congenital hyperinsulinism?

"Symptoms usually begin in the first 18 months of life, with excessive hunger and hypoglycaemia as the predominant symptoms. They can be treated and managed with dietary adjustments, and injections of insulin." - Anonymous Online Contributor

Unverified Answer

What is congenital hyperinsulinism?

"The term 'congenital' relates to the early onset of the condition, from birth. The symptoms of this disorder (the triad of hypoglycaemia, polyuria/polydipsia and failure to thrive) are often present from birth and may not be appreciated by the patient or the parents even at a later stage, and it may be misdiagnosed. A history of polyhydramnios or polyhydramnios-like symptoms from the first month of life leads to a diagnosis of congenital hyperinsulinism. Early surgery may prevent irreversible neurological damage, especially cerebral palsy. Genetic counselling is usually part of the pre-natal diagnosis of hyperinsulinism. © 2014 John Wiley & Sons, Ltd." - Anonymous Online Contributor

Unverified Answer

Can congenital hyperinsulinism be cured?

"Since only one-half of children with CDHI were treated, this study demonstrates that even with definitive treatment, a significant number of patients develop glucose intolerance or Type 1 diabetes by the age of 24 years-a finding that should serve as a call to action of this rare but devastating condition. Even with definitive treatment, all CDHI patients must be followed closely for persistent hyperglycemia and development of diabetes. We speculate that even in mild forms of CDHI, glucose levels should be closely monitored." - Anonymous Online Contributor

Unverified Answer

What is fluorodopa f 18?

"[The (18)F]fluorodopa PET scan was sensitive in detecting congenital hyperinsulinism in the basal ganglia in our population and was less sensitive in detecting adenomas, particularly in patients with concomitant hypogonadism." - Anonymous Online Contributor

Unverified Answer

Is fluorodopa f 18 typically used in combination with any other treatments?

"The FDG PET scan appears to be a valid imaging modality for assessing treatment response. We did not find any evidence for the use of FDG PET in combination with any other treatment." - Anonymous Online Contributor

Unverified Answer

Does congenital hyperinsulinism run in families?

"Although no single phenotype is associated with increased risk for developing CHI, this is most likely the result of a complex inheritance pattern including both gene-environment interactions and modifier variants." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of congenital hyperinsulinism?

"Primary congenital hyperinsulinism is a complex disorder affecting multiple systems, including the central nervous system and the autonomic system, leading to an increased risk for life-threatening consequences. The hyperinsulinism in HH is thought to be the consequence of a dysfunctional pathway of insulin signaling leading to excess glucose production by beta cells. The insulin resistance underlying HH can be caused by genetic considerations or by an undefined insult during childhood. At least in a minority of the cases, hyperinsulinemia cannot justify the symptoms, even though the primary source in these patients is most frequently attributable to a malfunction of the insulin signaling pathway." - Anonymous Online Contributor

Unverified Answer

What is the latest research for congenital hyperinsulinism?

"[This list is based on data presented in the Congenital Hyperinsulinism Working Group of the Expert Panel of the European Community Reference Group for Hyperinsulinaemia and Insulin Resistance in Childhood of the European Society of Pediatric Endocrinology and Diabetes (ESPEND) and of the European Society of Endocrinology(http://esend.eurohpg.org/en/profiles_of_research.aspx?Id=33)]\n- A novel mutation is identified in the alpha (SUR1A) and beta (INSR and SLC2A11) subunits of the insulin receptor (IR)." - Anonymous Online Contributor

Unverified Answer

What does fluorodopa f 18 usually treat?

"This is the first study to report the use of FDG PET in the identification and localization of lesions in patients presenting with neonatal and infantile CHHI. With the imaging, we not only provide valuable information for the clinical diagnosis and follow-up of CHHI, but also confirm the clinical diagnosis of pheochromocytoma which would prove to be the most appropriate initial management." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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