Current Clinical Trial Landscape of Gastroenteropancreatic Neuroendocrine Tumors: A New Era of Landmark Trials
Open Access
- 17 September 2025
- journal article
- review article
- Published by MDPI AG in Journal of Clinical Medicine
- Vol. 14 (18) , 6522
Abstract
Gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are a heterogeneous group of malignancies characterized by varying degrees of aggressiveness and clinical behavior. Despite advancements in treatment, including targeted therapies such as sunitinib and everolimus, peptide receptor radionuclide therapy with [177Lu]Lu-DOTATATE, and chemotherapy regimens like capecitabine plus temozolomide, the advanced GEP-NETs remain largely incurable. The limited efficacy of current treatments highlights the urgent need for novel therapeutic strategies. Recent years have seen a rise in several landmark clinical trials aimed at exploring new agents and combinations to improve patient outcomes in GEP-NETs. This literature review focuses on the ongoing clinical trials that hold promise for advancing the treatment landscape of GEP-NETs. We include some industry- and cooperative group-sponsored, phase II-III, randomized, and comparative trials in our review. We analyze the design, rationale, objectives, and preliminary findings of these trials, with a particular emphasis on those with pending results that may offer new insights into potential therapeutic targets. By examining these trials, we aim to provide a comprehensive overview of the evolving strategies in the management of GEP-NETs and underscore the importance of continued research innovation in addressing the challenges posed by the heterogeneity of GEP-NETs and in the pursuit of more effective and potentially curative treatment options.Keywords
Funding Information
- University of Arizona, Division of Hematology and Medical Oncology
This publication has 66 references indexed in Scilit:
- Established and novel circulating neuroendocrine tumor biomarkers for diagnostic, predictive and prognostic useBest Practice & Research Clinical Endocrinology & Metabolism, 2023
- Overview of the 2022 WHO Classification of Neuroendocrine NeoplasmsEndocrine Pathology, 2022
- A multigenomic liquid biopsy biomarker for neuroendocrine tumor disease outperforms CgA and has surgical and clinical utilityAnnals of Oncology, 2021
- Functioning and nonfunctioning pNENsCurrent Opinion in Endocrine and Metabolic Research, 2021
- Broadening horizons with 225Ac-DOTATATE targeted alpha therapy for gastroenteropancreatic neuroendocrine tumour patients stable or refractory to 177Lu-DOTATATE PRRT: first clinical experience on the efficacy and safetyEuropean Journal of Nuclear Medicine and Molecular Imaging, 2019
- Prognostic factors of patients with gastroenteropancreatic neuroendocrine neoplasmsThe Kaohsiung Journal of Medical Sciences, 2018
- Nothing But NET: A Review of Neuroendocrine Tumors and CarcinomasNeoplasia, 2017
- Identification of primary tumors in patients presenting with metastatic gastroenteropancreatic neuroendocrine tumorsSurgery, 2017
- Capecitabine and temozolomide (CAPTEM) for metastatic, well-differentiated neuroendocrine cancers: The Pancreas Center at Columbia University experienceCancer Chemotherapy and Pharmacology, 2013
- Somatostatin and Its Receptor FamilyFrontiers in Neuroendocrinology, 1999