Efficacy and Safety of Rapamycin in the Treatment of Crohn's Disease-related Stricture
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Response rate
研究概览
简要总结
Crohn's disease （CD） with stenosis has limited therapeutic options and with high surgical rate. The present clinical trial aims to evaluate the efficacy and safety of rapamycin in the treatment of stricturing Crohn's Disease.
详细描述
Management of Crohn's disease （CD） with stenosis is challenging and often requires endoscopic dilatation or surgical resection of the strictured bowel. Sirolimus (rapamycin), a macrocyclic antibiotic with immunosuppressive and antineoplastic properties, has been reported as promising rescue therapy for refractory CD. This study aims to evaluate the use of sirolimus for stricturing Crohn's Disease. Patients in this study will receive a continuous dosing schedule of oral sirolimus 2mg daily for six months. Clinical responses were defined as the ability to tolerate the regular diet with vegetable fiber combined with a reduction of ≥ 75% in overall target score and a score of less than two points for each item.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Chinese patients (≥18 years of age) with a documented definite diagnosis of CD;
- •the presence of a clinically symptomatic stricture;
- •strictures confirmed by endoscopy (passage of the endoscope with resistance or not traversable) or imaging (CT enterography (CTE) or MR enterography (MRE).
排除标准
- •Patients who were pregnant, diagnosed with intestinal perforation, complete intestinal obstruction, any signs of dysplasia or malignancy, or use of anti-tumor necrosis factor (TNF) in the last three months;
- •Patients who were not followed up between the inception of medication and any other subsequent treatments.
研究组 & 干预措施
Upper gastrointestinal strictures
Patients with upper gastrointestinal strictures were treated with rapamycin (2mg/day, Sirolimus, Roche) for at least six months.
干预措施: Rapamycin (Drug)
Lower gastrointestinal strictures
Patients with lower gastrointestinal strictures were treated with rapamycin (2mg/day, Sirolimus, Roche) for at least six months.
干预措施: Rapamycin (Drug)
结局指标
主要结局
Response rate
时间窗: up to 24 weeks
Response was defined as the following criteria: (a) the ability to tolerate a normal diet (vegetable fiber), with a reduction of ≥ 75% in overall baseline target score and sub-score ≤ 2 (Table S1); (b) no need for ED or surgery; (c) no severe adverse events or any other reasons leading to rapamycin withdrawal.
次要结局
- The rate of surgery or ED after rapamycin(through study completion, an average of 3 years)
- Adverse events(through study completion, an average of 3 years)
研究者
Faming Zhang
Associate professor, Gastroenterology
The Second Hospital of Nanjing Medical University