Crohn's Diseases
Conditions
Keywords
rapamycin, stricture, fibrosis
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.
Interventions
DRUGRapamycin
Participants with gastrointestinal strictures were treated with rapamycin 2mg daily at least six months.
Sponsors
The Second Hospital of Nanjing Medical University
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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) |
| 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. |
Countries
China
Contacts
Primary ContactFaming Zhang, MD,PhD
Outcome results
None listed