18F-Fluoro-L- DOPA PET Imaging for the Detection and Localization of Focal Congenital Hyperinsulinism
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 50
- 试验地点
- 2
- 主要终点
- Accuracy of Imaging
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 0 Days 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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 was
- •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.
排除标准
- •Treatment with other, third-line, medications for hyperinsulinism (nifedipine, glucagon).
- •Patients with hepatic or renal insufficiency.
研究组 & 干预措施
Single Arm,
Patients with clinically diagnosed congenital hyperinsulinism
干预措施: 18F-Fluoro Dopa Imaging (Drug)
结局指标
主要结局
Accuracy of Imaging
时间窗: 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
Sensitivity of Imaging
时间窗: 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
次要结局
- Semiquantitative Imaging Assessment(four weeks)
研究者
Miguel Pampaloni
Associate Professor of Radiology & Medicine
University of California, San Francisco