Original ResearchClinical—Alimentary TractSafety and Efficacy of Antigen-Specific Regulatory T-Cell Therapy for Patients With Refractory Crohn's Disease
Section snippets
Patients
This phase 1/2a, exploratory, uncontrolled, and open-label clinical trial was conducted at 6 centers in France between 2008 and 2011. The study protocol and amendments were approved by the appropriate independent ethics committee and national regulatory authority (Eudract no. 2006-004712-44). The study was performed in accordance with Good Clinical Practice guidelines. An independent data safety monitoring board was appointed to independently evaluate the ethical aspect and ensure patients'
Patient Dispositions, Demographics, and Baseline Disease Characteristics
Of 36 patients screened, 29 were included in the study and had blood collected for ova-Treg production (Figure 1A). At the pre-entry visit, 9 patients no longer met the inclusion criteria and were excluded from the study. The remaining 20 patients were treated and evaluated for safety and efficacy assessments and represented the ITT population. The initial design considered 3 patients per cohort, with 9 subjects planned for the last cohort with the highest dose. This distribution was amended,
Discussion
The CATS1 study is the first-in-human study of antigen-specific Tregs, a somatic cell medicinal product in clinical development for the treatment of inflammatory bowel disease. This open-label, escalating-dose study was conducted in patients with active CD refractory to all available agents for whom the unmet medical need was the greatest. The present results showed good tolerability and potential signals of efficacy for ova-Tregs.
Several approaches for the treatment of CD are currently in
Acknowledgments
The authors thank all of the patients in the CATS1 study, and all medical and nursing staff at the participating centers for their help; the trial data safety monitoring board: Professor Philippe Marteau, Professor Bruno Quesnel, and the late Professor Dominique Emilie (chair); the late Professor Marc Lémann for his significant contribution during the conduct of the study as a friend and principal investigator at Hospital St Louis (Paris, France); and Professor Pierre Tiberghien and Dr Pascal
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Conflicts of interest The authors disclose the following: Pierre Desreumaux, Matthieu Allez, and Jean-Frédéric Colombel are consultants for TxCell, and Arnaud Foussat, Valérie Brun, Hervé Bastian, Nathalie Belmonte, Virginie Neveu, Nathalie Clerget-Chossat, and Miguel Forte are employed by TxCell. Laurent Beaugerie, Xavier Hébuterne, Yoram Bouhnik, Maria Nachury, Michel Ticchioni, Agnès Duchange, and Patricia Morel-Mandrino disclose no conflicts of interest.
Funding Supported by TxCell SA.