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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Upper gastrointestinal strictures | Experimental | Patients with upper gastrointestinal strictures were treated with rapamycin (2mg/day, Sirolimus, Roche) for at least six months. |
|
| Lower gastrointestinal strictures | Experimental | Patients with lower gastrointestinal strictures were treated with rapamycin (2mg/day, Sirolimus, Roche) for at least six months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Rapamycin | Drug | Participants with gastrointestinal strictures were treated with rapamycin 2mg daily at least six months. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Response rate | 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. | up to 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Adverse events | New onset of symptoms and the exacerbation of previous symptoms were recorded as adverse events (AEs) | through study completion, an average of 3 years |
| The rate of surgery or ED after rapamycin |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Faming Zhang, MD,PhD | Contact | 086-25-58509883 | fzhang@njmu.edu.cn |
| Name | Affiliation | Role |
|---|---|---|
| Faming Zhang, MD,PhD | The Second Hospital of Nanjing Medical University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Medical Center for Digestive Diseases, The Second Affiliated Hospital of Nanjing Medical University | Recruiting | Nanjing | Jiangsu | 210011 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25240477 | Background | 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. | |
| 18719139 | Background | 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. |
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| ID | Term |
|---|---|
| D003424 | Crohn Disease |
| D003251 | Constriction, Pathologic |
| D005355 | Fibrosis |
| ID | Term |
|---|---|
| D015212 | Inflammatory Bowel Diseases |
| D005759 | Gastroenteritis |
| D005767 | Gastrointestinal Diseases |
| D004066 | Digestive System Diseases |
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| ID | Term |
|---|---|
| D020123 | Sirolimus |
| ID | Term |
|---|---|
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
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|
For long-term treatment outcomes, the primary endpoint was the rate of surgery or ED after rapamycin.
| through study completion, an average of 3 years |
| D007410 | Intestinal Diseases |
| D020763 | Pathological Conditions, Anatomical |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D010335 | Pathologic Processes |