Analysis of Clinical Trial Screen Failures in Inflammatory Bowel Diseases (IBD): Real World Results from the International Organization for the study of IBD.
Vieujean, Sophie; Lindsay, James O; D'Amico, Ferdinandoet al.
[en] [en] BACKGROUND: Recruitment for randomized controlled trials (RCTs) in IBD have substantially dropped over time. This study aimed to assess reasons why IBD patients are not included in sponsored multicenter phase IIb-III RCTs.
METHODS: All IOIBD members (n=58) were invited to participate. We divided barriers to participation as follow: 1) reasons patients with active IBD were not deemed appropriate for a RCT; 2) reasons qualified patients did not wish to participate; 3) reasons for screen failure (SF) in patients agreeing to participate. We assess those 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 10 centers across the world, were included in the prospective study. A RCT has not been proposed to 65 of them (mainly due to eligibility criteria). Of the 41 patients to whom a RCT was offered, 8 refused (mainly due to reluctance to receive placebo) and 28 agreed to participate. Among these 28 patients, 5 failed their screening and 23 were finally included in a RCT. A total of 107 patients (61 male (57%), 67 CD (62.6%)), from 13 centers worldwide, were included in our retrospective study of SFs. The main reason was insufficient disease activity.
CONCLUSION: This first multicenter study analyzing reasons for non-enrollment in IBD RCTs shown that we lose patients at each step. Eligibility criteria, the risk of placebo assignment and insufficient disease activity were part of the main barriers.
Disciplines :
Gastroenterology & hepatology
Author, co-author :
Vieujean, Sophie ; Centre Hospitalier Universitaire de Liège - CHU > > Service de gastroentérologie, hépatologie, onco. digestive
Lindsay, James O; Blizard Institute, Barts and the London School of Medicine and Dentistry, London, UK, Department of Gastroenterology, The Royal London Hospital, Barts Health NHS Trust, London,UK
D'Amico, Ferdinando; Department of Gastroenterology and Endoscopy, IRCCS San Raffaele Hospital and Vita-Salute San Raffaele University, Milan, Italy ; Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, Milan, Italy
Ahuja, Vineet ; Department of Gastroenterology, All India Institute of Medical Sciences, New Delhi, India
Silverberg, Mark S; Toronto Immune and Digestive Health Institute, Toronto, Canada
Sood, Ajit ; Department of Gastroenterology, Dayanand Medical College and Hospital, Ludhiāna, Punjab, India
Yamamoto-Furusho, Jesus K ; Inflammatory Bowel Disease Clinic, Department of Gastroenterology, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Tlalpa, Mexico
Nagahori, Masakazu; Department of Gastroenterology and Hepatology, Tokyo Medical and Dental University, Tokyo, Japan
Watanabe, Mamoru; Advanced Research Institute, Tokyo Medical and Dental University, Tokyo, Japan
Koutroubakis, Ioannis E; Department of Gastroenterology, University Hospital of Heraklion, Heraklion, Crete, Greece
Foteinogiannopoulou, Kalliopi ; Department of Gastroenterology, University Hospital of Heraklion, Heraklion, Crete, Greece
Avni Biron, Irit ; Division of Gastroenterology, Rabin Medical Center, Petah Tikva, Israel, and Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
Walsh, Alissa; Translational Gastroenterology Unit, John Radcliffe Hospital, Oxford University Hospitals NHS Foundation Trust and NIHR Biomedical Research Centre, Oxford, United Kingdom
Outtier, An; Department of Gastroenterology and Hepatology, University Hospitals Leuven, KU Leuven, Leuven, Belgium
Nordestgaard, Rie Louise Møller; Gastrounit, Medical Division, Copenhagen University Hospital - Amager and Hvidovre, Hvidovre, Denmark
Abreu, Maria T; Division of Gastroenterology, Department of Medicine, University of Miami Miller School of Medicine, Miami, FL, USA
Dubinsky, Marla; Division of Pediatric Gastroenterology and Nutrition, Icahn School of Medicine, Mount Sinai, New York, New York
Siegel, Corey ; Inflammatory Bowel Disease Center, Section of Gastroenterology and Hepatology, Dartmouth-Hitchcock Medical Center, Lebanon, New Hampshire
Louis, Edouard ; Centre Hospitalier Universitaire de Liège - CHU > > Service de gastroentérologie, hépatologie, onco. digestive
Dotan, Iris; Division of Gastroenterology, Rabin Medical Center, Petah Tikva, Israel, and Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
Reinisch, Walter ; Department of Internal Medicine III, Medical University of Vienna, Vienna, Austria
Danese, Silvio ; Department of Gastroenterology and Endoscopy, IRCCS San Raffaele Hospital and Vita-Salute San Raffaele University, Milan, Italy
Rubin, David T; University of Chicago Medicine Inflammatory Bowel Disease Center, Chicago, IL, USA
Peyrin-Biroulet, Laurent; Department of Gastroenterology, Nancy University Hospital, F-54500 Vandœuvre-lès-Nancy, France ; INFINY Institute, Nancy University Hospital, F-54500 Vandœuvre-lès-Nancy, France ; FHU-CURE, Nancy University Hospital, F-54500 Vandœuvre-lès-Nancy, France ; Groupe Hospitalier privé Ambroise Paré - Hartmann, Paris IBD center, 92200 Neuilly sur Seine, France ; Division of Gastroenterology and Hepatology, McGill University Health Centre, Montreal, Quebec, Canada
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