Clinical Trial: Non-functioning Pancreatic Neuroendocrine Tumors in MEN1: Somatostatin Analogs Versus NO Treatment

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: Non-functioning Pancreatic Neuroendocrine Tumors (NF-pNETs) in Multiple Endocrine Neoplasia Type 1 (MEN1) Treated With Somatostatin Analogs (SA) Versus NO Treatment - a Pr

Brief Summary:

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.

Detailed Summary:

1. 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).

   As shown recently in a retrospective study of 40 patients with early-stage MEN1 duodeno-pancreatic NETs, treatment with SAs was safe and effective, resulting in long-time suppression of tumor and hormonal activity and 10% objective response. The authors suggest to start therapy with SAs early on in patients with MEN1-related NETs (12). Apart from this clinical study, there is one case report on SAs for MEN-1-related insulinoma (13).

   MEN1 is an orphan disease (ORPHA652).

2. 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.