Evolving landscape of clinical trials in gastroenteropancreatic neuroendocrine neoplasms in the past two decades
Open accessAbstract
Background: Gastroenteropancreatic neuroendocrine neoplasms (GEP-NENs) are heterogenous malignancies that require well-designed trials to develop effective management strategies. This cross-sectional study aimed to illustrate the current landscape of clinical trials in GEP-NENs to provide insights for future research. Materials and methods: We reviewed all clinical trials registered on ClinicalTrials.gov between 1 January 2000 and 31 December 2021 with GEP-NEN in the ‘condition or disease’ field. Results: We included 206 eligible trials. Most trials enrolled less than 50 patients (59.8%) and were sponsored by institutions other than government or industry (67.0%). Most trials were conducted in high-income countries (86.6%) and countries located in Europe (30.1%) or Northern America (29.6%). The overall result reporting rates of GEP-NEN trials was 41.4%, and the median time from primary completion to result reporting was 101 months. Characteristics that improved the reporting of results included larger sample size, tumor differentiation specification for inclusion, progression-free survival as primary endpoint, industry sponsorship, and multicenter or multinational participation (all P < 0.05). Compared with trials registered between 2000 and 2011 ( n = 28), trials registered between 2012 and 2021 ( n = 178) were more likely to specify the Ki-67 index for inclusion (68.0% vs 35.7%, P = 0.002) and to be conducted outside Europe or Northern America (16.4% vs 3.7%, P = 0.02), while the sample size and the sponsorship did not change significantly. Conclusions: Novel management options have been explored for GEP-NENs with more specific inclusion criteria during the past two decades. More efforts are needed to promote international collaborations in clinical trials and enhance timely result dissemination.
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Lutetium-177 dotatate (Lutathera) targets the somatostatin receptor overexpressed in neuroendocrine tumors.
“Several successful examples of radiopharmaceuticals utilizing active targeting include Lutetium-177 dotatate (Lutathera), which targets the somatostatin receptor overexpressed in neuroendocrine tumors.”
From Radiopharmaceuticals: navigating the frontier of precision medicine and therapeutic innovation (2024) · cites this paper as [21]
The Discussion (page unavailable) says neuroendocrine tumors express somatostatin receptors, and the References list a trial of 177Lu-Dotatate for midgut neuroendocrine tumors. The paper does not state that 177Lu-Dotatate itself targets those receptors or identify it as Lutathera; its explicit example of a receptor-bound radioactive peptide concerns other isotopes. (LLM, conf=0.84)
Experimental — accuracy not yet measuredA phase III clinical trial of Lutathera demonstrated improved progression-free survival and overall response rates compared to standard therapy in gastroenteropancreatic neuroendocrine tumors.
“Approved by the FDA in 2018 for treating gastroenteropancreatic neuroendocrine tumors, a phase III clinical trial demonstrated improved progression-free survival and overall response rates compared to standard therapy [21].”
From Radiopharmaceuticals: navigating the frontier of precision medicine and therapeutic innovation (2024) · cites this paper as [21]
The Introduction identifies peptide receptor radionuclide therapy as a treatment used to inhibit tumor growth, and the References list a phase 3 trial of 177Lu-Dotatate in midgut neuroendocrine tumors (page numbers unavailable). The paper does not report that trial’s progression-free survival or response-rate results versus standard therapy, or state the claimed 2018 FDA approval. (LLM, conf=0.82)
Experimental — accuracy not yet measuredActive targeting is effective in tailoring radiation therapy to specific cancer cells, marking a significant advancement in precision medicine.
“These examples showcase the effectiveness of active targeting in tailoring radiation therapy to specific cancer cells, marking a significant advancement in precision medicine.”
From Radiopharmaceuticals: navigating the frontier of precision medicine and therapeutic innovation (2024) · cites this paper as [21]
The Introduction says peptide receptor radionuclide therapy (PRRT) is used to inhibit tumor growth, and the Discussion calls PRRT a milestone in systemic treatment of gastroenteropancreatic neuroendocrine neoplasms (pages not provided). The paper does not establish that active targeting tailors radiation to specific cancer cells or demonstrate that effectiveness across cancers generally. (LLM, conf=0.81)
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Radiopharmaceuticals: navigating the frontier of precision medicine and therapeutic innovation
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“Approved by the FDA in 2018 for treating gastroenteropancreatic neuroendocrine tumors, a phase III clinical trial demonstrated improved progression-free survival and overall response rates compared to standard therapy [21].”
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- Authors
- Yang, K., Li, J., Cheng, Y., & Bai, C.
- Affiliations
- Chinese Academy of Medical Sciences & Peking Union Medical College (CN)
- Peking University Cancer Hospital (CN)
- Corresponding author
- Cheng, Yuejuan
- Bai, Chunmei
- Venue
- Endocrine Connections
- Publisher
- Bioscientifica
- Published
- 2023-02-01
- Volume
- 12
- Issue
- 4
- ISSN
- 2049-3614 (ISSN-L)
- PMC
- PMC10083666
- OpenAlex
- W4318753750
- Cited by
- 4
- References
- 50
- Citations by year
- 2026: 1 · 2025: 1 · 2024: 1 · 2023: 1
- FWCI
- 0.80
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Endocrine Connections (publishedVersion, cc-by-nc)
- PubMed Central (publishedVersion, cc-by-nc)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Topics
- Health Sciences › Medicine › Epidemiology › Neuroendocrine Tumor Research Advances
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmacology › Pharmaceutical industry and healthcare
- Health Sciences › Medicine › Gastroenterology › Gastrointestinal Tumor Research and Treatment
- Keywords
- gastroenteropancreatic neuroendocrine tumors, clinical trial landscape, ClinicalTrials.gov, clinical trial registration, result reporting, trial sponsorship, trial inclusion criteria, Ki-67 proliferation index, tumor differentiation, sample size, international cooperation
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