TY - JOUR
T1 - Analysis of Clinical Trial Screen Failures in Inflammatory Bowel Diseases [IBD]
T2 - Real World Results from the International Organization for the study of IBD
AU - Vieujean, Sophie
AU - Lindsay, James O.
AU - D’Amico, Ferdinando
AU - Ahuja, Vineet
AU - Silverberg, Mark S.
AU - Sood, Ajit
AU - Yamamoto-Furusho, Jesus K.
AU - Nagahori, Masakazu
AU - Watanabe, Mamoru
AU - Koutroubakis, Ioannis E.
AU - Foteinogiannopoulou, Kalliopi
AU - Biron, Irit Avni
AU - Walsh, Alissa
AU - Outtier, An
AU - Nordestgaard, Rie Louise Møller
AU - Abreu, Maria T.
AU - Dubinsky, Marla
AU - Siegel, Corey
AU - Louis, Edouard
AU - Dotan, Iris
AU - Reinisch, Walter
AU - Danese, Silvio
AU - Rubin, David T.
AU - Peyrin-Biroulet, Laurent
N1 - Publisher Copyright:
© The Author(s) 2023. Published by Oxford University Press on behalf of European Crohn’s and Colitis Organisation.
PY - 2024/4/1
Y1 - 2024/4/1
N2 - Background: Recruitment for randomized controlled trials [RCTs] in inflammatory bowel diseases [IBD] has substantially dropped over time. This study aimed to assess reasons why IBD patients are not included in sponsored multicentre phase IIb–III RCTs. Methods: All IOIBD members [n = 58] were invited to participate. We divided barriers to participation as follows: [1] reasons patients with active IBD were not deemed appropriate for an RCT; [2] reasons qualified patients did not wish to participate; and [3] reasons for screen failure [SF] in patients agreeing to participate. We assess these in a 4-week prospective study including, consecutively, all patients with symptomatic disease for whom a treatment change was required. In addition, we performed a 6-month retrospective study to further evaluate reasons for SF. Results: A total of 106 patients (60 male [56.6%], 63 Crohn’s disease [CD] [59.4%]), from ten centres across the world, were included in the prospective study. An RCT has not been proposed to 65 of them [mainly due to eligibility criteria]. Of the 41 patients to whom an RCT was offered, eight refused [mainly due to reluctance to receive placebo] and 28 agreed to participate. Among these 28 patients, five failed their screening and 23 were finally included in an RCT. A total of 107 patients (61 male [57%], 67 CD [62.6%]), from 13 centres worldwide, were included in our retrospective study of SFs. The main reason was insufficient disease activity. Conclusion: This first multicentre study analysing reasons for non-enrolment in IBD RCTs shows that we lose patients at each step. Eligibility criteria, the risk of placebo assignment, and insufficient disease activity were part of the main barriers.
AB - Background: Recruitment for randomized controlled trials [RCTs] in inflammatory bowel diseases [IBD] has substantially dropped over time. This study aimed to assess reasons why IBD patients are not included in sponsored multicentre phase IIb–III RCTs. Methods: All IOIBD members [n = 58] were invited to participate. We divided barriers to participation as follows: [1] reasons patients with active IBD were not deemed appropriate for an RCT; [2] reasons qualified patients did not wish to participate; and [3] reasons for screen failure [SF] in patients agreeing to participate. We assess these in a 4-week prospective study including, consecutively, all patients with symptomatic disease for whom a treatment change was required. In addition, we performed a 6-month retrospective study to further evaluate reasons for SF. Results: A total of 106 patients (60 male [56.6%], 63 Crohn’s disease [CD] [59.4%]), from ten centres across the world, were included in the prospective study. An RCT has not been proposed to 65 of them [mainly due to eligibility criteria]. Of the 41 patients to whom an RCT was offered, eight refused [mainly due to reluctance to receive placebo] and 28 agreed to participate. Among these 28 patients, five failed their screening and 23 were finally included in an RCT. A total of 107 patients (61 male [57%], 67 CD [62.6%]), from 13 centres worldwide, were included in our retrospective study of SFs. The main reason was insufficient disease activity. Conclusion: This first multicentre study analysing reasons for non-enrolment in IBD RCTs shows that we lose patients at each step. Eligibility criteria, the risk of placebo assignment, and insufficient disease activity were part of the main barriers.
KW - Inflammatory bowel disease
KW - non-enrolment
KW - randomized controlled trials
KW - screen failure
UR - http://www.scopus.com/inward/record.url?scp=85191106164&partnerID=8YFLogxK
U2 - 10.1093/ecco-jcc/jjad180
DO - 10.1093/ecco-jcc/jjad180
M3 - Article
C2 - 37864829
AN - SCOPUS:85191106164
SN - 1873-9946
VL - 18
SP - 548
EP - 559
JO - Journal of Crohn's and Colitis
JF - Journal of Crohn's and Colitis
IS - 4
ER -