RT Journal Article
T1 ENSAT registry-based randomized clinical trials for adrenocortical carcinoma.
A1 Crona, Joakim
A1 Baudin, Eric
A1 Terzolo, Massimo
A1 Chrisoulidou, Alexandra
A1 Angelousi, Anna
A1 Ronchi, Cristina L
A1 Oliveira, Cristina Lamas
A1 Nieveen van Dijkum, Els J M
A1 Ceccato, Filippo
A1 Borson-Chazot, Françoise
A1 Reimondo, Giuseppe
A1 Tiberi, Guido A M
A1 Ettaieb, Hester
A1 Kiriakopoulos, Andreas
A1 Canu, Letizia
A1 Kastelan, Darko
A1 Osher, Esthr
A1 Yiannakopoulou, Eugenia
A1 Arnaldi, Giorgio
A1 Assié, Guillaume
A1 Paiva, Isabel
A1 Bourdeau, Isabelle
A1 Newell-Price, John
A1 Nowak, Karolina M
A1 Romero, M Tous
A1 De Martino, Maria Cristina
A1 Bugalho, Maria João
A1 Sherlock, Mark
A1 Vantyghem, Marie-Christine
A1 Dennedy, Michael Conall
A1 Loli, Paula
A1 Rodien, Patrice
A1 Feelders, Richard
A1 de Krijger, Ronald
A1 Van Slycke, Sam
A1 Aylwin, Simon
A1 Morelli, Valentina
A1 Vroonen, Laurent
A1 Shafigullina, Zulfiya
A1 Bancos, Irina
A1 Trofimiuk-Müldner, Małgorzata
A1 Quinkler, Marcus
A1 Luconi, Michaela
A1 Kroiss, Matthias
A1 Naruse, Mitsuhide
A1 Igaz, Peter
A1 Mihai, Radu
A1 Della Casa, Silvia
A1 Berruti, Alfredo
A1 Fassnacht, Martin
A1 Beuschlein, Felix
AB 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.
YR 2021
FD 2021
LK http://hdl.handle.net/10668/16571
UL http://hdl.handle.net/10668/16571
LA en
DS RISalud
RD Apr 12, 2025