Clinical trial NCT02675153: To Evaluate the Efficacy and Safety of Rapamycin for Crohn's Disease-related Stricture
Official title: Efficacy and Safety of Rapamycin in the Treatment of Crohn's Disease-related Stricture
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Crohn's Diseases
Keywords: rapamycin
stricture
fibrosis
Lead sponsor: The Second Hospital of Nanjing Medical University
Sponsor class: OTHER
First posted: 2016-02-05
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Response rate
timeFrame: up to 24 weeks
Secondary outcomes:
measure: Adverse events
timeFrame: through study completion, an average of 3 years
measure: The rate of surgery or ED after rapamycin
timeFrame: through study completion, an average of 3 years
Other outcomes:
Locations:
city: Nanjing
country: China
facility: Medical Center for Digestive Diseases, The Second Affiliated Hospital of Nanjing Medical University
state: Jiangsu
Study officials:
affiliation: The Second Hospital of Nanjing Medical University
name: Faming Zhang, MD,PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Mutalib M, Borrelli O, Blackstock S, Kiparissi F, Elawad M, Shah N, Lindley K. The use of sirolimus (rapamycin) in the management of refractory inflammatory bowel disease in children. J Crohns Colitis. 2014 Dec;8(12):1730-4. doi: 10.1016/j.crohns.2014.08.014. Epub 2014 Sep 18.
citation: Massey DC, Bredin F, Parkes M. Use of sirolimus (rapamycin) to treat refractory Crohn's disease. Gut. 2008 Sep;57(9):1294-6. doi: 10.1136/gut.2008.157297.