Searchable abstracts of presentations at key conferences in endocrinology
Previous issue | Volume 98 | NANETS2023



Trials In Progress

ea0098t1 | Trials In Progress | NANETS2023

ACTION-1: A randomized Phase Ib/3 trial of RYZ101 compared with SoC in SSTR+ well-differentiated GEP-NET with progression following Lu-177 SSA

Hope Thomas A. , Halperin Daniel , Strosberg Jonathan , Jacene Heather , E.T. Tesselaar Margot , Kunz Pamela L. , Ferreira Denis , Li Joanne , Ma Kimberly , Rearden Jessica , Moran Susan , Singh Simron

Background: Well-differentiated gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are commonly characterized by high-density expression of somatostatin receptors (SSTRs), which can be targeted by radiopharmaceutical therapy (RPT) via radiolabeled somatostatin analogues (SSAs). RYZ101 (225Ac-DOTATATE) is a first-in-class, highly potent alpha-emitting RPT being developed for the treatment of SSTR+ solid tumors. Alpha-particles (such as those emitted by 225<...

ea0098t2 | Trials In Progress | NANETS2023

Phase 2 study of nab-sirolimus in patients with well-differentiated and advanced/metastatic neuroendocrine tumors of the gastrointestinal tract, lung, or pancreas

Demeure, MD Michael J. , Cohn, MD Allen L. , Seery, MD Tara , Ding, MS, MA Li , Aziz, MD Usman , Navarro, MD Willis , Paulson, MD Scott

Background: Neuroendocrine tumors (NETs; ~2% of all malignancies) commonly arise from the GI tract, pancreas, and lung and often present with metastatic disease. The PI3K/Akt/mTOR pathway is implicated in the pathogenesis and progression of NETs. The oral mTOR inhibitor (mTORi), everolimus, is approved for treatment of patients with NETs of the GI tract, lung, or pancreas. However, due to the rarity and heterogeneity, nonspecific clinical symptoms, and unique indolent biology,...

ea0098t3 | Trials In Progress | NANETS2023

Phase 1/2 trial of Pb-212-VMT-alpha-NET in GI neuroendocrine tumors and pheochromocytoma/paraganglioma previously treated with radioligand therapy

Lin Frank I. , Del Rivero Jaydira , Carrasquillo Jorge , Shamis Inna , Zou Joy , Turkbey Baris , Klubo Joanna , Mena Esther , Lindenberg Liza , Chen Clara , Herscovitch Peter , Millo Corina , Pacak Karel

Background: Metastatic GI Neuroendocrine Tumors (GI-NET) and pheochromocytoma/paraganglioma (PPGL) are are tumors which overexpresses somatostatin receptors (SSTR) and can be treated with targeted radioligand therapy (RLT) such as Lu-177-DOTATATE. However, despite demonstrated clinical efficacy at stabilizing tumor growth, durable response is very rare and almost all patients inevitably progresses at some time after treatment. Good systemic therapy options after beta-emiting R...

ea0098t4 | Trials In Progress | NANETS2023

Phase 2, multicenter, open-label basket trial of nab-sirolimus for malignant solid tumors harboring pathogenic inactivating alterations in TSC1/2 (PRECISION I)

Iyer, MD Gopa , Demeure, MD Michael J. , Ding, MS, MA Li , Schmid, PhD Anita N. , Navarro, MD Willis , Kwiatkowski, MD, PhD David J. , Rodon Ahnert, MD, PhD Jordi

Background: nab-Sirolimus, approved in the US for patients with advanced malignant PEComa, is a novel albumin-bound mTOR inhibitor (mTORi) that inhibits the mTOR pathway via suppression of the mTORC1 complex. When TSC1 or TSC2 is inactivated via mutation or loss, the mTOR pathway may be aberrantly activated. TSC1 and TSC2 alterations occur in a range of common cancers. Clinically, in the AMPECT exploratory analysis of nab-sirolimus in advanced malignant PEComa (NCT02494570), 8...

ea0098t5 | Trials In Progress | NANETS2023

Phase 3 LEVEL trial of 177Lu-edotreotide vs everolimus in patients with advanced neuroendocrine tumors of lung or thymic origin (GETNE-T2217)

Capdevila Jaume , Fazio Nicola , Alvarez Rosa , Ansquer Catherine , Baldari Sergio , Baudin Eric , Benavent Marta , Benini Lavinia , Beron Amandine , Berruti Alfredo , Cingarlini Sara , del Olmo-Garcia Maribel , Deshayes Emmanuel , Garcia-Alvarez Alejandro , Garcia-Carbonero Rocio , Haissaguerre Magalie , Hernando Jorge , Anido Herranz Urbano , Jimenez-Fonseca Paula , Lepage Come , Llana Belen , Molina-Cerrillo Javier , Panzuto Francesco , Pubul Virginia , Sansovini Maddalena , Tafuto Salvatore , Taieb David , Teule Alex , Versari Annibale , Villacampa Guillermo , Walter Thomas

Background: Everolimus is the only approved drug for patients with advanced bronchopulmonary neuroendocrine tumors (NET), and there is an urgent unmet need for alternative treatments. Retrospective data for peptide receptor radionuclide therapy (PRRT) have demonstrated promising activity in somatostatin receptor (SST)-positive lung NET. This study aims to investigate the clinical efficacy, safety, and patient-reported outcomes when 177Lu-edotreotide is used to treat...

ea0098t6 | Trials In Progress | NANETS2023

Genetic profiling in the randomized controlled phase 3 COMPOSE trial of 177Lu-edotreotide for well-differentiated aggressive grade 2/3 gastroenteropancreatic neuroendocrine tumors

Halfdanarson Thorvardur R. , Capdevila Jaume , Halperin Daniel M. , Herrmann Ken , Kong Grace , Mailman Josh , Reidy-Lagunes Diane , Srirajaskanthan Raj , Sierras Cristina , Rotger Amanda

Background: Targeted radionuclide therapies (TRTs) have changed the treatment paradigm of neuroendocrine tumors (NET) and are expected to be widely available for patients with various gastroenteropancreatic (GEP)-NET phenotypes. However, while they have great potential, TRT-based therapeutic strategies for high-grade GEP-NET demonstrate variable outcomes, and there is a current lack of tools to identify patients who are likely to respond to TRT. To address this need, the rando...

ea0098t7 | Trials In Progress | NANETS2023

Phase 1 trial of Pb-212-VMT-alpha-NET in select metastatic or inoperable somatostatin receptor positive tumors

Lin Frank I. , Del Rivero Jaydira , Thomas Anish , Srinivasan Ramaprasad , Charalampos Floudas , Carrasquillo Jorge , Shamis Inna , Zou Joy , Turkbey Baris , Mena Esther , Lindenberg Liza , Chen Clara , Herscovitch Peter , Millo Corina , Pacak Karel

Background: Somatostatin receptors (SSTR) is overexpressed in a number of different tumors, including GI Neuroendocrine Tumors (GI-NET), Pheochromocytoma/Paraganglioma (PPGL), small cell lung cancer (SCLC), renal cell carcinoma (RCC), and certain head and neck cancers (H&N) such as olfactory neuroblastoma. It has been demonstrated that these SSTR-expressing tumors can be treated with beta-emitting radioligand therapy (RLT) that binds to SSTR such as Lu-177-DOTATATE. Howeve...

ea0098t8 | Trials In Progress | NANETS2023

Status of the ongoing SORENTO clinical trial: Assessing efficacy and safety of high-exposure octreotide subcutaneous depot in patients with GEP-NET

Singh Simron , Capdevila Jaume , Ang Chan Jennifer , W de Herder Wouter , Grozinsky-Glasberg Simona , Halfdanarson Thorvardur , M Halperin Daniel , Mailman Josh , Hellstrom Lisa , Svedberg Agneta , Tiberg Fredrik , Ferone Diego

Acknowledgements: This study was funded by Camurus AB. Medical writing support was provided by Costello Medical and funded by Camurus AB.Background: Somatostatin receptor ligands (SRLs) are first-line standard of care therapies for gastroenteropancreatic neuroendocrine tumors (GEP-NET), showing efficacy in tumor/symptom control with an established safety profile. However, disease progression usually occurs despite standard-dose SRL treatment, requiring m...

ea0098t9 | Trials In Progress | NANETS2023

Phase 1-2 trial of vesicular stomatitis virus expressing human interferon-β and NIS (VSV-IFNβ-NIS), with ipilimumab and nivolumab, in patients with neuroendocrine carcinoma

McGarrah Patrick W. , Naik Shruthi , Halfdanarson Thorvardur R. , Shanshal Mohamed , Whye Peng Kah , Russell Stephen J. , Adjei Alex A. , Molina Julian R.

Background: Poorly differentiated neuroendocrine carcinoma (NEC) is an aggressive malignancy comprising both pulmonary and extrapulmonary primary sites. NEC includes both small cell lung cancer (SCLC) and large cell neuroendocrine carcinoma (LCNEC). The optimal systemic therapy beyond first line platinum and etoposide is not established. There is a critical need to improve upon the median survival in the second line, as most patients do not survive more than 6 months. The effi...

ea0098t10 | Trials In Progress | NANETS2023

Phase Ib trial of cabozantinib (Cabometyx®) combined with Lu-177 DOTATATE radioligand therapy in patients with advanced, somatostatin receptor positive NETs.

F Kennecke MD, MHA, FRCPC Hagen , Yamasaki, RN Lena , Kasi, MD, MPH Anup , Herz Katherine , Mittra MD Erik S.

Background: Combination peptide receptor radionuclide therapy (PRRT) with the multikinase inhibitor cabozantinib may result in enhanced tumor response and improved intratumoral delivery of Lu-177 DOTATATE by normalization of tumor vasculature through VEGFR inhibition.Methods: In a phase Ib trial, patients with advanced somatostatin receptor (SSTR) positive, G1-3 neuroendocrine tumors (NETs) with a Krenning score of >2 are treated with 4 x 8 week cycl...

ea0098t11 | Trials In Progress | NANETS2023

NET RETREAT: a Phase II Study of 177Lutetium-Dotatate Retreatment vs. Everolimus in Metastatic/Unresectable Midgut NET

Chauhan Aman , O'Callaghan Chris , Myrehaug Sten , Bodei Lisa , Kunz Pamela , Dasari Arvind , Strosberg Jonathan , Alexander Stefanie , Cheung Winson , Singh Simron

Background: 177Lu-DOTATATE is an FDA and Health Canada-approved treatment option for metastatic, progressive GEPNET patients. 177Lu-DOTATATE is now often considered the treatment of choice for small bowel/midgut patients who have progressed on somatostatin analogs (SSA). Despite 177Lu- DOTATATE’s impressive disease stabilization, many patients will eventually progress. Progression after prior use of PRRT does not necessarily render these tumors resistant to future PRRT tr...

ea0098t12 | Trials In Progress | NANETS2023

Neuroendocrine tumors AI-based clinical trial search tool eases clinical trial discovery for patients and health care professionals

Mailman Josh , Ralic Danielle , del Rivero MD Jaydira , Gericke MD Germo , Halfdanarson MD Thorvardur R. , Herrmann MD Ken , Hollander Ronald , Albert Fisher Jr. MD George

Background: Finding relevant neuroendocrine tumor (NET) trials remains a challenge for patients and healthcare professionals (HCPs).’s taxonomy associates many conditions with NETs, complicating the discovery process. As of 6/30/23 listed 700 recruiting, phase 1-3 interventional trials using the terms Neuroendocrine Tumors, Pheochromocytoma, and Paraganglioma. Many of these trials are not relevant for patients with NETs. Even with sev...