The Value of a Central Image Management System (CIMS) in the Conduct of Randomized Controlled Trials of Therapy for Ulcerative Colitis (UC)

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Purpose: Increased reliance has been placed on the use of endoscopic scoring as inclusion criteria and outcome measures in clinical trials for UC. Central scoring (CS), in distinction to site investigator scoring (SIS), has potential for reducing both bias and measurement variability. Methods: We utilized data from a placebo-controlled randomized trial of Asacol®, an 800 mg formulation of mesalamine, conducted in patients with mildly-to-moderately active UC. Eligible patients had a Mayo Clinic endoscopy sub-score ≥2 with a total Mayo Clinic score of 4-10 points (inclusive). Patients were randomized 1:1 to treatment with Asacol® 4.8 g/day or placebo for 10 weeks. Outcomes were assessed at weeks 6 and 10. We performed posthoc exploratory efficacy analyses comparing clinical and endoscopic outcomes at week 6 in SIS (ITT) and CS-defined endoscopically eligible populations. We also compared week 6 outcomes in CS-eligible patients using site versus central scoring. Results: A total of 281 patients comprised the SIS (ITT) population. Of these, 194 (69%) were considered eligible by CS and were included from CS-based analyses. See Table 1 for week 6 results. The effect size (percent difference Asacol® minus placebo) was consistently greater for all outcomes in the CS-versus SIS (ITT)-defined population. Placebo rates were uniformly greater in the SIS (ITT) population due to the inclusion of patients with low endoscopic disease activity as judged by the central reader. However, within the CS-defined population, no difference in effect size was observed between CS- and SIS-based outcomes at week 6.Table: [1454] Week 6 efficacy analysesConclusion: CS evaluation of endoscopic eligibility criteria has the potential to improve efficiency of randomized controlled trials of induction therapy in UC by minimizing inclusion of patients with low endoscopic disease activity who increase placebo rates. No difference was detected in effect size generated by CS and SIS when the eligible population was defined by CS. The rights to Asacol®, including the rights to the trademark Asacol®, are not owned by Tillotts Pharma AG in any of the following countries: Switzerland, USA, United Kingdom, Canada, Italy, Belgium, the Netherlands, and Luxembourg. Disclosure: Dr. Feagan - Advisory Board Member Tillotts. Dr. Sandborn - Advisory Board Member: Tillotts. Dr. D'Haens - Received speaking honoraria: Tillotts. Dr. Rutgeerts - Advisory Board Member: Tillotts. Dr. Munkholm - Advisory Board Member: Tillotts. Dr. Mittmann - Consultant: Tillotts. Dr. Gilgen - Employee: Tillotts. This research was supported by an industry grant from Tillotts.

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