Division of Gastroenterology and Hepatology, Department of Internal Medicine, Toho University Sakura Medical Center, Chiba, Japan.
Medical Affairs Division, Immunology and Infectioous Disease Department, Janssen Pharmaceutical K.K, Chiyoda-ku, Japan.
BMJ Open. 2022 May 4;12(5):e060081. doi: 10.1136/bmjopen-2021-060081.
Ulcerative colitis (UC) is an idiopathic, chronic inflammatory disease of the large intestine. Ustekinumab is a monoclonal antibody against the p40 subunit of interleukin-12 and interleukin-23 and has proven efficacy in inducing and maintaining remission in adult patients with moderate-to-severe UC. In the Symptom Improvement of ulceRative colitis after an Induction dose of Ustekinumab study, we will document the initial treatment response (daily patient-reported outcomes for 8 weeks from first infusion) and treatment patterns of patients wih UC receiving an induction dose of ustekinumab in the real-world setting in Japan. We will also investigate the relationship between the treatment response at week 8 and early indicators of response and determine patient factors that may define the appropriate dosing interval for maintenance therapy.
For this single-arm, prospective observational study at 24 centres in Japan with a follow-up period of 16/20 weeks, we aim to recruit 140 patients with moderate-to-severe UC between July 2021 and July 2022. All surveys will be conducted in Japanese and patient-reported outcomes relating to rectal bleeding, stool frequency, abdominal pain, nocturnal diarrhoea, tenesmus and perception of UC symptoms will be recorded using a smartphone application, where the patients can enter their initial response to ustekinumab induction therapy on a daily basis. Dosing intervals and the reasons for selecting this interval, and concomitant medications taken during treatment with ustekinumab will be collected by a physician questionnaire at the end of the study. On completion of primary end point (8-week patient-reported outcomes) data collection, results will be reported sequentially.
The study has been approved by the ethics committee of each facility involved and the Institutional Review Board of the non-profit organisation MINS.
UMIN000043753, NCT04963725.
溃疡性结肠炎（UC）是一种特发性、慢性大肠炎症性疾病。乌司奴单抗是一种针对白细胞介素-12 和白细胞介素-23 的 p40 亚单位的单克隆抗体，已被证明可有效诱导和维持中重度 UC 成年患者的缓解。在乌司奴单抗诱导剂量治疗溃疡性结肠炎症状改善研究中，我们将记录患者的初始治疗反应（首次输注后 8 周的每日患者报告结局）和接受乌司奴单抗诱导剂量治疗的 UC 患者的治疗模式。我们还将研究第 8 周的治疗反应与早期反应指标之间的关系，并确定可能定义维持治疗适当给药间隔的患者因素。
这是一项在日本 24 个中心进行的单臂、前瞻性观察研究，随访期为 16/20 周，我们计划在 2021 年 7 月至 2022 年 7 月期间招募 140 名中重度 UC 患者。所有调查都将用日语进行，并且使用智能手机应用程序记录与直肠出血、粪便频率、腹痛、夜间腹泻、里急后重和 UC 症状感知相关的患者报告结局，患者可以在此应用程序上每日记录他们对乌司奴单抗诱导治疗的初始反应。在研究结束时，医生问卷将收集剂量间隔和选择该间隔的原因，以及乌司奴单抗治疗期间使用的伴随药物。完成主要终点（8 周患者报告结局）数据收集后，将按顺序报告结果。
该研究已获得参与机构的伦理委员会和非营利组织 MINS 的机构审查委员会的批准。
UMIN000043753，NCT04963725。
