ENSAT Registry-Based Randomized Clinical Trials for Adrenocortical Carcinoma.
Joakim Crona
(1)
,
Eric Baudin
(2)
,
Massimo Terzolo
,
Alexandra Chrisoulidou
,
Anna Angelousi
,
Cristina L. Ronchi
(3)
,
Cristina Lamas
,
Els Nieveen van Dijkum
(4)
,
Filippo Ceccato
,
Françoise Borson-Chazot
(5)
,
Guiseppe Reimondo
(6)
,
Guido A. Tiberi
(7)
,
Hester Ettaieb
,
Andreas Kiriakopoulos
,
Letizia Canu
(8)
,
Darko Kastelan
,
Esthr Osher
(9)
,
Eugenia Yiannakopoulou
(10)
,
Giorgio Arnaldi
(11)
,
Guillaume Assie
(12)
,
Isabel Paiva
,
Isabelle Bourdeau
,
John Newell-Price
,
Karolina M. Nowak
,
M. Tous Romero
,
Maria Cristina de Martino
(13)
,
Maria Joao Bugalho
,
Mark Sherlock
,
Marie-Christine Vantyghem
(14)
,
Michael Conall Dennedy
(15)
,
Paula Loli
,
Patrice Rodien
(16)
,
Richard A. Feelders
,
Ronald R. de Krijger
,
Sam van Slycke
,
Simon Aylwin
,
Valentina Morelli
,
Laurent Vroonen
,
Zulfiya Shafigullina
,
Irina Bancos
,
Malgorzata Trofimiuk-Müldner
,
Marcus Quinkler
,
Michaela Luconi
(8)
,
Matthias Kroiss
(17)
,
Mitsuhide Naruse
,
Peter Igaz
,
Radu Mihai
(18)
,
Silvia Della Casa
,
Alfredo Berruti
,
Martin Fassnacht
(17)
,
Felix Beuschlein
,
Anna Angelousi
1
Uppsala University
2 Université Paris-Saclay
3 University of Birmingham [Birmingham]
4 UvA - University of Amsterdam [Amsterdam] = Universiteit van Amsterdam
5 UCBL - Université Claude Bernard Lyon 1
6 UNITO - Università degli studi di Torino = University of Turin
7 UniBs - Università degli Studi di Brescia = University of Brescia
8 UniFI - Università degli Studi di Firenze = University of Florence = Université de Florence
9 TAU - Tel Aviv University
10 UNIWA - University of West Attica [Athens]
11 UNIVPM - Polytechnic University of Marche / Università Politecnica delle Marche
12 IC UM3 (UMR 8104 / U1016) - Institut Cochin
13 University of Naples Federico II = Università degli studi di Napoli Federico II
14 CHRU Lille - Centre Hospitalier Régional Universitaire [CHU Lille]
15 NUI Galway - National University of Ireland [Galway]
16 CHU Angers - Centre Hospitalier Universitaire d'Angers
17 JMU - Julius-Maximilians-Universität Würzburg = University of Würzburg [Würsburg, Germany]
18 University of Oxford
2 Université Paris-Saclay
3 University of Birmingham [Birmingham]
4 UvA - University of Amsterdam [Amsterdam] = Universiteit van Amsterdam
5 UCBL - Université Claude Bernard Lyon 1
6 UNITO - Università degli studi di Torino = University of Turin
7 UniBs - Università degli Studi di Brescia = University of Brescia
8 UniFI - Università degli Studi di Firenze = University of Florence = Université de Florence
9 TAU - Tel Aviv University
10 UNIWA - University of West Attica [Athens]
11 UNIVPM - Polytechnic University of Marche / Università Politecnica delle Marche
12 IC UM3 (UMR 8104 / U1016) - Institut Cochin
13 University of Naples Federico II = Università degli studi di Napoli Federico II
14 CHRU Lille - Centre Hospitalier Régional Universitaire [CHU Lille]
15 NUI Galway - National University of Ireland [Galway]
16 CHU Angers - Centre Hospitalier Universitaire d'Angers
17 JMU - Julius-Maximilians-Universität Würzburg = University of Würzburg [Würsburg, Germany]
18 University of Oxford
Massimo Terzolo
- Fonction : Auteur
Alexandra Chrisoulidou
- Fonction : Auteur
Anna Angelousi
- Fonction : Auteur
Cristina Lamas
- Fonction : Auteur
Filippo Ceccato
- Fonction : Auteur
Hester Ettaieb
- Fonction : Auteur
Andreas Kiriakopoulos
- Fonction : Auteur
Darko Kastelan
- Fonction : Auteur
Isabel Paiva
- Fonction : Auteur
Isabelle Bourdeau
- Fonction : Auteur
John Newell-Price
- Fonction : Auteur
Karolina M. Nowak
- Fonction : Auteur
M. Tous Romero
- Fonction : Auteur
Maria Joao Bugalho
- Fonction : Auteur
Mark Sherlock
- Fonction : Auteur
Paula Loli
- Fonction : Auteur
Richard A. Feelders
- Fonction : Auteur
Ronald R. de Krijger
- Fonction : Auteur
Sam van Slycke
- Fonction : Auteur
Simon Aylwin
- Fonction : Auteur
Valentina Morelli
- Fonction : Auteur
Laurent Vroonen
- Fonction : Auteur
Zulfiya Shafigullina
- Fonction : Auteur
Irina Bancos
- Fonction : Auteur
Malgorzata Trofimiuk-Müldner
- Fonction : Auteur
Marcus Quinkler
- Fonction : Auteur
Mitsuhide Naruse
- Fonction : Auteur
Peter Igaz
- Fonction : Auteur
Silvia Della Casa
- Fonction : Auteur
Alfredo Berruti
- Fonction : Auteur
Felix Beuschlein
- Fonction : Auteur
Anna Angelousi
- Fonction : Auteur
Résumé
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.