ENSAT registry-based randomized clinical trials for adrenocortical carcinoma

Joakim Crona, Eric Baudin, Massimo Terzolo, Alexandra Chrisoulidou, Anna Angelousi, Cristina L. Ronchi, Cristina Lamas Oliveira, Els J.M. Nieveen van Dijkum, Filippo Ceccato, Françoise Borson-Chazot, Giuseppe Reimondo, Guido A.M. Tiberi, Hester Ettaieb, Andreas Kiriakopoulos, Canu Letizia, Darko Kastelan, Esthr Osher, Eugenia Yiannakopoulou, Giorgio Arnaldi, Guillaume AssiéIsabel Paiva, Isabelle Bourdeau, John Newell-Price, Karolina M. Nowak, M. Tous Romero, Maria Cristina de Martino, Maria João Bugalho, Mark Sherlock, Marie Christine Vantyghem, Michael Conall Dennedy, Paula Loli, Patrice Rodien, Richard Feelders, Ronald de Krijger, Sam van Slycke, Simon Aylwin, Valentina Morelli, Laurent Vroonen, Zulfiya Shafigullina, Irina Bancos, Małgorzata Trofimiuk-Müldner, Marcus Quinkler, Michaela Luconi, Matthias Kroiss, Mitsuhide Naruse, Peter Igaz, Radu Mihai, Silvia della Casa, Alfredo Berruti, Martin Fassnacht, Felix Beuschlein

Onderzoeksoutput: Bijdrage aan tijdschriftArtikel recenserenpeer review

7 Citaten (Scopus)

Samenvatting

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.

Originele taal-2Engels
Pagina's (van-tot)R51-R59
TijdschriftEuropean Journal of Endocrinology
Volume184
Nummer van het tijdschrift2
DOI's
StatusGepubliceerd - feb. 2021

Vingerafdruk

Duik in de onderzoeksthema's van 'ENSAT registry-based randomized clinical trials for adrenocortical carcinoma'. Samen vormen ze een unieke vingerafdruk.

Citeer dit