To Evaluate the Efficacy and Safety of Rapamycin for Crohn's Disease-related Stricture

Not Applicable

• Conditions: Crohn's Diseases
• Interventions: Drug: Rapamycin

• Registration Number: NCT02675153
• Lead Sponsor: The Second Hospital of Nanjing Medical University

Brief Summary

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.

Detailed Description

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.

Study Timeline

• Study Start: 2015-04-01
• Study First Submitted: 2016-01-21
• Study First Submitted QC: 2016-02-02
• Study First Posted: 2016-02-05
• Status Verified: 2020-10
• Primary Completion: 2020-10
• Study Completion: 2020-10
• Last Update Submitted: 2020-10-02
• Last Update Posted: 2020-10-08

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1. Chinese patients (≥18 years of age) with a documented definite diagnosis of CD;
2. the presence of a clinically symptomatic stricture;
3. strictures confirmed by endoscopy (passage of the endoscope with resistance or not traversable) or imaging (CT enterography (CTE) or MR enterography (MRE).

Exclusion Criteria

1. 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;
2. Patients who were not followed up between the inception of medication and any other subsequent treatments.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Upper gastrointestinal strictures	Rapamycin	Patients with upper gastrointestinal strictures were treated with rapamycin (2mg/day, Sirolimus, Roche) for at least six months.
Lower gastrointestinal strictures	Rapamycin	Patients with lower gastrointestinal strictures were treated with rapamycin (2mg/day, Sirolimus, Roche) for at least six months.

Primary Outcome Measures

Name	Time	Method
Response rate	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.

Secondary Outcome Measures

Name	Time	Method
The rate of surgery or ED after rapamycin	through study completion, an average of 3 years	For long-term treatment outcomes, the primary endpoint was the rate of surgery or ED after rapamycin.
Adverse events	through study completion, an average of 3 years	New onset of symptoms and the exacerbation of previous symptoms were recorded as adverse events (AEs)

Trial Locations

Locations (1)

Medical Center for Digestive Diseases, The Second Affiliated Hospital of Nanjing Medical University; 🇨🇳 Nanjing, Jiangsu, China