Non-functioning Pancreatic Neuroendocrine Tumors (NF-pNETs) in Multiple Endocrine Neoplasia Type 1 (MEN1) Treated With Somatostatin Analogs (SA) Versus NO Treatment - a Prospective, Randomized, Controlled Multicenter Study
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 180
- 主要终点
- Growth rate of the tumor in mm
研究概览
简要总结
A.Background
More than 90% of patients with multiple endocrine neoplasia type 1 (MEN1) develop multiple pancreatic neuroendocrine tumors (pNETs). These tumors are the most common cause for premature death in MEN1.
While functioning pNETs must be treated to reduce or cure hormonal excess, the procedures for non-functioning pNETs are yet under discussion. Treatment ranges from watchful waiting to subtotal and total pancreatectomy. The latter may represent an "overtreatment", resulting in general complications and diabetic metabolic status.
The effect of somatostatin analogues (SAs) has shown promising results with regard to progression of non-functioning duodeno-pancreatic NETs. Treatment with SAs is highly safe and effective, resulting in long-time suppression of tumor growth.
B. Aim
In this study of MEN1 patients with non-functioning pNETs, the benefits of somatostatin analogs" (SAs; group 1) compared to "no treatment" (group 2) will be analyzed with regard to progression (tumor growth; development of new [functioning and non-functioning] neuroendocrine tumors and regional/distant metastasis).
C. Implementation
Patients will either receive Somatostatin Analogs (SAs) or no treatment. The observation period will be 60 months. The increase of tumor size and development of new tumors or metastasis will be monitored.
详细描述
- Introduction
1.1 Background
Due to the genetic background of the disease, every single neuroendocrine cell of the pancreas is a potential progenitor of neuroendocrine tumors (NETs). More than 90% of patients with multiple endocrine neoplasia type 1 (MEN1) develop multiple pancreatic neuroendocrine tumors (pNETs) "viewable" by transgastric endosonography and/or cross sectional and/or functional imaging. These tumors are the most common cause for premature death in MEN1 (1, 2).
While functioning pNETs are to be treated to reduce or cure hormonal excess, the strategies of addressing non-functioning (NF) pNETs are under discussion. Treatment ranges from "watchful waiting" to subtotal or total pancreatectomy (3-6). The latter may prove to be an "overtreatment" resulting in diabetic metabolic status and subsequently in general long-term complications.
Somatostatin analogs (SAs) have shown promising results with regard to progression-free survival in patients with metastatic NETs of the midgut (9-11).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Verified MEN1 syndrome by molecular genetics (known mutation)
- •Non-functioning pNET
- •Largest ("leading") pancreatic tumor with ≤20 mm in diameter and (if present) one small tumor <15 mm in diameter as reference lesion
- •G1 or G2 (Ki-67 ≤ 10%) according to endoscopic ultrasound/fine-needle aspiration (EUS/FNA) acquired by 19-gauge needle
- •Functional imaging: Ga68-DOTA-conjugated peptide positron emission tomography (PET) computed tomography (CT) or preferably Ga68-DOTA-conjugated peptide magnetic resonance imaging (MRI)
- •Tumor(s) limited to the pancreas (N0, M0)
排除标准
- •Functioning tumor - hormone excess
- •Neuroendocrine carcinoma (G3)
- •Metastatic disease (N1, M1)
研究组 & 干预措施
Somatostatin-Analog
A long acting somatostatin analog will be applied.
干预措施: Somatostatin-Analog (Drug)
结局指标
主要结局
Growth rate of the tumor in mm
时间窗: 5 years
The growth rate of the leading lesion (≥20mm in diameter) will be radiologically controlled in six-monthly intervals. Growth rate will be compared between the groups.
次要结局
- Documentation of lymph node and/or distant metastases(5 years)
- Documentation of new tumors(5 years)
研究者
Dr. Andreas Selberherr
M.D.
Medical University of Vienna