ENSAT registry-based randomized clinical trials for adrenocortical carcinoma

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 intro...

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Published in:European journal of endocrinology Vol. 184; no. 2; p. R51
Main Authors: Crona, Joakim, Baudin, Eric, Terzolo, Massimo, Chrisoulidou, Alexandra, Angelousi, Anna, Ronchi, Cristina L, Oliveira, Cristina Lamas, Nieveen van Dijkum, Els J M, Ceccato, Filippo, Borson-Chazot, Françoise, Reimondo, Giuseppe, Tiberi, Guido A M, Ettaieb, Hester, Kiriakopoulos, Andreas, Canu, Letizia, Kastelan, Darko, Osher, Esthr, Yiannakopoulou, Eugenia, Arnaldi, Giorgio, Assié, Guillaume, Paiva, Isabel, Bourdeau, Isabelle, Newell-Price, John, Nowak, Karolina M, Romero, M Tous, De Martino, Maria Cristina, Bugalho, Maria João, Sherlock, Mark, Vantyghem, Marie-Christine, Dennedy, Michael Conall, Loli, Paula, Rodien, Patrice, Feelders, Richard, de Krijger, Ronald, Van Slycke, Sam, Aylwin, Simon, Morelli, Valentina, Vroonen, Laurent, Shafigullina, Zulfiya, Bancos, Irina, Trofimiuk-Müldner, Małgorzata, Quinkler, Marcus, Luconi, Michaela, Kroiss, Matthias, Naruse, Mitsuhide, Igaz, Peter, Mihai, Radu, Della Casa, Silvia, Berruti, Alfredo, Fassnacht, Martin, Beuschlein, Felix
Format: Journal Article
Language:English
Published: England 01.02.2021
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ISSN:1479-683X, 1479-683X
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Abstract 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.
AbstractList 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.
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.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.
Author Borson-Chazot, Françoise
Crona, Joakim
Ceccato, Filippo
Aylwin, Simon
Luconi, Michaela
Bourdeau, Isabelle
Paiva, Isabel
Kroiss, Matthias
Osher, Esthr
De Martino, Maria Cristina
Loli, Paula
Mihai, Radu
Arnaldi, Giorgio
Quinkler, Marcus
Bugalho, Maria João
Feelders, Richard
Sherlock, Mark
Reimondo, Giuseppe
Della Casa, Silvia
Baudin, Eric
Fassnacht, Martin
Van Slycke, Sam
Assié, Guillaume
Beuschlein, Felix
Tiberi, Guido A M
Yiannakopoulou, Eugenia
Bancos, Irina
Rodien, Patrice
Oliveira, Cristina Lamas
Ettaieb, Hester
Chrisoulidou, Alexandra
Morelli, Valentina
Nieveen van Dijkum, Els J M
de Krijger, Ronald
Berruti, Alfredo
Ronchi, Cristina L
Trofimiuk-Müldner, Małgorzata
Nowak, Karolina M
Dennedy, Michael Conall
Terzolo, Massimo
Igaz, Peter
Angelousi, Anna
Naruse, Mitsuhide
Newell-Price, John
Kiriakopoulos, Andreas
Shafigullina, Zulfiya
Kastelan, Darko
Romero, M Tous
Vantyghem, Marie-Christine
Canu, Letizia
Vroonen, Laurent
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Snippet Adrenocortical carcinoma (ACC) is an orphan disease lacking effective systemic treatment options. The low incidence of the disease and high cost of clinical...
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SubjectTerms Adrenal Cortex Neoplasms - diagnosis
Adrenal Cortex Neoplasms - epidemiology
Adrenal Cortex Neoplasms - therapy
Adrenocortical Carcinoma - diagnosis
Adrenocortical Carcinoma - epidemiology
Adrenocortical Carcinoma - therapy
Endocrinology - organization & administration
Endocrinology - standards
Europe
Evidence-Based Medicine - organization & administration
Evidence-Based Medicine - standards
Evidence-Based Medicine - trends
Humans
Randomized Controlled Trials as Topic - methods
Randomized Controlled Trials as Topic - standards
Randomized Controlled Trials as Topic - statistics & numerical data
Registries
Social Networking
Title ENSAT registry-based randomized clinical trials for adrenocortical carcinoma
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Volume 184
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