NCT03000296

Autologous Unselected Hematopoietic Stem Cell Transplantation for Refractory Crohn's Disease

• Status: Unknown
• Phase: N/A
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Beneficência Portuguesa de São Paulo · Academic / Other
• Sex: All
• Age: 14 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This study evaluates the safety and clinical benefits of a therapeutic approach using the cyclophosphamide (Cy) + thymoglobulin® (ATG) + granulocyte colony-stimulating factor (G-CSF) conditioning regimen followed by autologous hematopoietic stem cell transplantation (HSCT) rescue in the treatment of refractory Crohn's disease. Adverse events, and clinical and endoscopic conditions will be assessed at different short and long-term time points.

Detailed description

Crohn's disease (CD) is a chronic, refractory inflammatory bowel disease that affects the entire digestive tract associated with intestinal and extra intestinal manifestations or other autoimmune diseases. Conventional therapy for Crohn's disease includes anti-inflammatory, immunosuppressant and/or biologic drugs/corticosteroids. This treatment benefits the majority of patients. However, a proportion of patients fail to achieve complete and long-term disease control and often require multiple intestinal surgeries with a risk of developing short bowel syndrome. Hematopoietic stem cell transplantation (HSCT) has been proposed to cause lymphoablation and reset of the immune system as an alternative strategy to induce long-term disease control in this high-risk population. This study enrolled Crohn's disease patients not responsive to conventional therapy. Initially safety and the clinical outcome will be evaluated. The selected patients will be admitted to the bone marrow transplant (BMT) unit for the mobilization regimen using cyclophosphamide (Cy - 60 mg/kg) and G-CSF (10 mcg/kg/day) from the 5th day after Cy administration until harvesting progenitor cells from peripheral blood by leukapheresis. After seven days of rest, the conditioning regimen consists of Cy (200 mg/kg total dose for four days), rabbit antithymocyte globulin (6.5 mg/kg total dose for four days) and methylprednisolone (500 mg/day). The clinical course of patients with refractory Crohn´s disease will be evaluated to determine the efficacy of HSCT as a therapeutic tool including the adverse aspects of the procedure, clinical outcome and quality of life.

Conditions

• Crohn's Disease
• Inflammatory Bowel Diseases
• Gastroenteritis

Interventions

Type	Name	Description
PROCEDURE	Autologous Hematopoietic Stem Cell Transplantation	Hematopoietic stem cell transplantation Lymphoablation followed by hematopoietic stem cell transplantation to rescue the immune system.

Timeline

• Start date: 2013-10-01
• Primary completion: 2017-12-01
• Completion: 2024-12-01
• First posted: 2016-12-22
• Last updated: 2021-01-15

Locations

1 site across 1 country: Brazil