ENSAT registry-based randomized clinical trials for adrenocortical carcinoma

J Crona, E Baudin, M Terzolo, A Chrisoulidou, A Angelousi, CL Ronchi, CL Oliveira, E van Dijkum, F Ceccato, F Borson-Chazot, G Reimondo, GAM Tiberi, H Ettaieb, A Kiriakopoulos, C Letizia, D Kastelan, E Osher, E Yiannakopoulou, G Arnaldi, G AssieI Paiva, I Bourdeau, J Newell-Price, KM Nowak, MT Romero, MC de Martino, MJ Bugalho, M Sherlock, MC Vantyghem, MC Dennedy, P Loli, P Rodien, R.A. Feelders, R de Krijger, S Van Slycke, S Aylwin, V Morelli, L Vroonen, Z Shafigullina, I Bancos, M Trofimiuk-Muldner, M Quinkler, M Luconi, M Kroiss, M Naruse, P Igaz, R Mihai, S Della Casa, A Berruti, M Fassnacht, F Beuschlein

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Adrenocortical carcinoma (ACC) is an orphan disease lacking effective systemic treatment options. The low incidence of the disease and high cost of clinical trials are major obstacles in the search for improved treatment strategies. As a novel approach, registry-based clinical trials have been introduced in clinical research, so allowing for significant cost reduction, but without compromising scientific benefit. Herein, we describe how the European Network for the Study of Adrenal Tumours (ENSAT) could transform its current registry into one fit for a clinical trial infrastructure. The rationale to perform randomized registry-based trials in ACC is outlined including an analysis of relevant limitations and challenges. We summarize a survey on this concept among ENSAT members who expressed a strong interest in the concept and rated its scientific potential as high. Legal aspects, including ethical approval of registry-based randomization were identified as potential obstacles. Finally, we describe three potential randomized registry-based clinical trials in an adjuvant setting and for advanced disease with a high potential to be executed within the framework of an advanced ENSAT registry. Thus we, therefore, provide the basis for future registry-based trials for ACC patients. This could ultimately provide proof-of-principle of how to perform more effective randomized trials for an orphan disease.

Original languageEnglish
Pages (from-to)R51-R59
JournalEuropean Journal of Endocrinology
Issue number2
Publication statusPublished - Feb 2021

Bibliographical note

Funding Information:
J C received funding from Region Uppsala, Cancerfonden and Tore Nilsons Stiftelse, F B and M F were supported by the German Research Foundation (DFG) project number 314061271 (CRC/TRR 205). M N received funding from Japan Agency for Medical Research and Development (AMED) (JP19ek0109352).

Funding Information:
J C received lecture honoraria from Novartis and educational honoraria from NET connect (funded by 阀psen). M K received institutional funding for a clinical trial of cabozantinib in advanced adrenocortical carcinoma from 阀psen. 阀 B reports advisory board participation with Corcept and HRA Pharma and consultancy fee with ClinCore outside the submitted work. F B C received honoraria for lecture or consultancy work from 阀psen, Novartis, Bayer, Esai and Novo Nordisk (unrelated to the present topic). S A has received lecture honoraria from HRA pharma. J N P has received research and consultancy grants from HRA Pharma, Novartis, Diurnal, Recordati (unrelated to the present topic). M N received lecture honoraria from DA 阃阀CH 阀 SANKYO Company, Ltd., and Kyowaki rin Co.Ltd. and consultancy grants from Ono Pharmaceutical Co. Ltd., and FUJ 阀F 阀LM R 阀 Pharma Co.,Ltd. (unrelated to the present topic). Guillaume Assie and Martin Fassnacht are Associate Editors of European Journal of Endocrinology. They were not involved in the editorial or peer review process of this paper, on which they are listed as authors.

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