{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,1]],"date-time":"2026-03-01T01:25:25Z","timestamp":1772328325011,"version":"3.50.1"},"reference-count":0,"publisher":"Oxford University Press (OUP)","issue":"2","content-domain":{"domain":["eje.bioscientifica.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2021,2]]},"abstract":"<jats:p>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.<\/jats:p>","DOI":"10.1530\/eje-20-0800","type":"journal-article","created":{"date-parts":[[2020,11,9]],"date-time":"2020-11-09T18:23:24Z","timestamp":1604946204000},"page":"R51-R59","update-policy":"https:\/\/doi.org\/10.1530\/crossmarkpolicy-7","source":"Crossref","is-referenced-by-count":14,"title":["ENSAT registry-based randomized clinical trials for adrenocortical carcinoma"],"prefix":"10.1093","volume":"184","author":[{"given":"Joakim","family":"Crona","sequence":"first","affiliation":[{"name":"1Department of Medical Sciences, Uppsala University, Uppsala, Sweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eric","family":"Baudin","sequence":"additional","affiliation":[{"name":"2Gustave Roussy, Universit\u00e9 Paris-Saclay, D\u00e9partement d\u2019imagerie, Service d\u2019oncologie endocrinienne, Villejuif, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Massimo","family":"Terzolo","sequence":"additional","affiliation":[{"name":"3Department of Clinical and Biological Sciences, University of Turin at San Luigi Hospital, Orbassano, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alexandra","family":"Chrisoulidou","sequence":"additional","affiliation":[{"name":"4Department of Endocrinology, Theagenio Cancer Hospital, Thessaloniki, Greece"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anna","family":"Angelousi","sequence":"additional","affiliation":[{"name":"51st Department of Internal Medicine, Unit of Endocrinology, National and Kapodistrian University of Athens, Laiko Hospital, Athens, Greece"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cristina L","family":"Ronchi","sequence":"additional","affiliation":[{"name":"6Institute of Metabolism and System Research, University of Birmingham, Birmingham, UK"},{"name":"7Division of Endocrinology and Diabetes, University Hospital of W\u00fcrzburg, W\u00fcrzburg, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cristina Lamas","family":"Oliveira","sequence":"additional","affiliation":[{"name":"8Endocrinology Department, Complejo Hospitalario Universitario de Albacete, Albacete, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Els J M","family":"Nieveen van Dijkum","sequence":"additional","affiliation":[{"name":"9Department of Surgery, Cancer Center Amsterdam, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Filippo","family":"Ceccato","sequence":"additional","affiliation":[{"name":"10Endocrinology Unit, Department of Medicine DIMED, University-Hospital of Padova, Padova, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fran\u00e7oise","family":"Borson-Chazot","sequence":"additional","affiliation":[{"name":"11Hospices Civils de Lyon, F\u00e9d\u00e9ration d\u2019Endocrinologie, Universit\u00e9 Claude Bernard Lyon 1, Lyon, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Giuseppe","family":"Reimondo","sequence":"additional","affiliation":[{"name":"12Internal Medicine, Department of Clinical and Biological Sciences, San Luigi Gonzaga Hospital, University of Turin, Orbassano, Turin, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Guido A M","family":"Tiberi","sequence":"additional","affiliation":[{"name":"13Surgical Clinic, Department of Clinical and Experimental Sciences, University of Brescia at ASST Spedali Civili, Brescia, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hester","family":"Ettaieb","sequence":"additional","affiliation":[{"name":"14Division of Endocrinology, Department of Internal Medicine, Maxima Medical Center, Eindhoven\/Veldhoven, The Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andreas","family":"Kiriakopoulos","sequence":"additional","affiliation":[{"name":"155th Surgical Clinic, \u2018Evgenidion Hospital\u2019 National and Kapodistrian University of Athens School of Medicine, Athens, Greece"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Letizia","family":"Canu","sequence":"additional","affiliation":[{"name":"16Department of Experimental and Clinical Biomedical Sciences, University of Florence, Florence, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Darko","family":"Kastelan","sequence":"additional","affiliation":[{"name":"17Department of Endocrinology, University Hospital Centre Zagreb, Zagreb, Croatia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Esthr","family":"Osher","sequence":"additional","affiliation":[{"name":"18Institute of Endocrinology, Metabolism and Hypertension, Tel Aviv-Sourasky Medical Center Israel, Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eugenia","family":"Yiannakopoulou","sequence":"additional","affiliation":[{"name":"19Department of Biomedical Sciences, Faculty of Health Sciences, University of West Attica, Athens, Greece"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Giorgio","family":"Arnaldi","sequence":"additional","affiliation":[{"name":"20Division of Endocrinology, Department of Clinical and Molecular Sciences, Polytechnic University of Marche, Ancona, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Guillaume","family":"Assi\u00e9","sequence":"additional","affiliation":[{"name":"21Universit\u00e9 de Paris, Institut Cochin, INSERM, CNRS, Paris, France"},{"name":"22Endocrinology, Assistance Publique H\u00f4pitaux de Paris, H\u00f4pital Cochin, Paris, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Isabel","family":"Paiva","sequence":"additional","affiliation":[{"name":"23Department os Endocrinology, Diabetes and Metabolism, Centro Hospitalar e Universit\u00e1rio de Coimbra, Coimbra, Portugal"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Isabelle","family":"Bourdeau","sequence":"additional","affiliation":[{"name":"24Division of Endocrinology, Department of Medicine, Research Center, Centre Hospitalier de l\u2019Universit\u00e9 de Montr\u00e9al (CHUM), Montreal, Qu\u00e9bec, Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"John","family":"Newell-Price","sequence":"additional","affiliation":[{"name":"25Department of Oncology and Metabolism, The Medical School University of Sheffield, Sheffield, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Karolina M","family":"Nowak","sequence":"additional","affiliation":[{"name":"26Department of Endocrinology, Centre of Postgraduate Medical Education, Bielanski Hospital, Warsaw, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M Tous","family":"Romero","sequence":"additional","affiliation":[{"name":"27UGC Endocrinolog\u00eda y Nutrici\u00f3n, Hospital Universitario Virgen Macarena, Seville, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria Cristina","family":"De Martino","sequence":"additional","affiliation":[{"name":"28Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Universit\u00e0 Federico II di Napoli, Naples, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria Jo\u00e3o","family":"Bugalho","sequence":"additional","affiliation":[{"name":"29Servi\u00e7o de Endocrinologia, Diabetes e Metabolismo, CHULN and Faculdade de Medicina da Universidade de Lisboa, Lisboa, Portugal"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mark","family":"Sherlock","sequence":"additional","affiliation":[{"name":"30Department of Endocrinology, Beaumont Hospital and the Royal College of Surgeons in Ireland, Dublin, Ireland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marie-Christine","family":"Vantyghem","sequence":"additional","affiliation":[{"name":"31Endocrinology, Diabetology, Metabolism and Nutrition Department, Lille University Hospital, Lille, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael Conall","family":"Dennedy","sequence":"additional","affiliation":[{"name":"32Department of Endocrinology & Diabetes Mellitus, c\/o Department of Medicine, Clinical Sciences Institute, National University of Ireland, Galway, Galway, Ireland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paula","family":"Loli","sequence":"additional","affiliation":[{"name":"33Ospedale Niguarda Ca\u2019 Granda, Endocrinology, Napoli, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Patrice","family":"Rodien","sequence":"additional","affiliation":[{"name":"34Service d\u2019Endocrinologie Diab\u00e9tologie et Nutrition, CHU d\u2019Angers, Angers Cedex 9, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Richard","family":"Feelders","sequence":"additional","affiliation":[{"name":"35Erasmus Medical Center, Division of Endocrinology, Department of Internal Medicine, Rotterdam, The Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ronald","family":"de Krijger","sequence":"additional","affiliation":[{"name":"36Princess M\u00e1xima Center for Pediatric Oncology, Utrecht, The Netherlands"},{"name":"37Department of Pathology, University Medical Center, Utrecht, The Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sam","family":"Van Slycke","sequence":"additional","affiliation":[{"name":"38General and Endocrine Surgery, OLV Hospital Aalst, Aalst, Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Simon","family":"Aylwin","sequence":"additional","affiliation":[{"name":"39King\u2019s College Hospital, London, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Valentina","family":"Morelli","sequence":"additional","affiliation":[{"name":"40Endocrinology Unit, Fondazione IRCCS Ca\u2019 Granda, Ospedale Maggiore Policlinico, Milan, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Laurent","family":"Vroonen","sequence":"additional","affiliation":[{"name":"41Department of Endocrinology, Centre Hospitalier Universitaire de Li\u00e8ge, Liege, Belgium"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zulfiya","family":"Shafigullina","sequence":"additional","affiliation":[{"name":"42Endocrinology Department, North-Western Medical University named after I.I.Mechnikov, Saint-Petersburg, Russia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Irina","family":"Bancos","sequence":"additional","affiliation":[{"name":"43Division of Endocrinology, Diabetes, Metabolism, and Nutrition, Mayo Clinic, Rochester, Minnesota, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ma\u0142gorzata","family":"Trofimiuk-M\u00fcldner","sequence":"additional","affiliation":[{"name":"44Department of Endocrinology, Jagiellonian University Medical College, Krak\u00f3w, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marcus","family":"Quinkler","sequence":"additional","affiliation":[{"name":"45Endocrinology in Charlottenburg, Berlin, 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Specialties, Radiological Sciences, and Public Health, Medical Oncology, University of Brescia at ASST Spedali Civili, Brescia, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Martin","family":"Fassnacht","sequence":"additional","affiliation":[{"name":"7Division of Endocrinology and Diabetes, University Hospital of W\u00fcrzburg, W\u00fcrzburg, Germany"},{"name":"46Comprehensive Cancer Center Mainfranken, University of W\u00fcrzburg, W\u00fcrzburg, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Felix","family":"Beuschlein","sequence":"additional","affiliation":[{"name":"53Klinik f\u00fcr Endokrinologie, Diabetologie und Klinische Ern\u00e4hrung, Universit\u00e4tsspital Z\u00fcrich, Z\u00fcrich, Switzerland"},{"name":"54Department of Endocrinology, Medizinische Klinik und Poliklinik IV, Klinikum der Universit\u00e4t M\u00fcnchen, Munich, 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