Autologous Unselected Hematopoietic Stem Cell Transplantation for Refractory Crohn's Disease (AutoChron)
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|ClinicalTrials.gov Identifier: NCT03000296|
Recruitment Status : Recruiting
First Posted : December 22, 2016
Last Update Posted : July 20, 2018
|Condition or disease||Intervention/treatment||Phase|
|Crohn's Disease Inflammatory Bowel Diseases Gastroenteritis||Procedure: Autologous Hematopoietic Stem Cell Transplantation||Not Applicable|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Autologous Unselected Hematopoietic Stem Cell Transplantation for Refractory Crohn's Disease|
|Study Start Date :||October 2013|
|Actual Primary Completion Date :||December 2017|
|Estimated Study Completion Date :||March 2020|
Experimental: Hematopoietic Stem Cell Transplantation
High doses immunosuppression Cyclophosphamide (200 mg/kg total dose for four days) and rabbit antithymocyte globulin (6.5 mg/kg total dose for four days) followed by unselected autologous hematopoietic stem cell transplantation rescue.
Procedure: Autologous Hematopoietic Stem Cell Transplantation
Hematopoietic stem cell transplantation Lymphoablation followed by hematopoietic stem cell transplantation to rescue the immune system.
Other Name: Bone Marrow Transplantation
- Safety of unselected autologous HSCT in refractory Crohn´s disease patients [ Time Frame: 12 months ]HHSCT safety will be analyzed by laboratory tests and treatment-related adverse events. Safety will be evaluated by treatment-related adverse events. All adverse events will be recorded in a standardized way and their relationship to the study protocol will be assessed at different short- and long-term time points.
- Crohn´s Disease Activity Index (CDAI) [ Time Frame: 12 months ]Duration of disease remission, defined as a CDAI ≤ 150, will be assessed at 1, 3, 6, 12 and 24 months after transplant.
- CRAIG Crohn´s Severity Score (CSS) [ Time Frame: 12 months ]The CRAIG CSS will be assessed at 1, 3, 6, 12 and 24 months after transplant.
- Inflammatory Bowel Disease Questionnaire (IBDQ) [ Time Frame: 24 months ]The IBDQ will be administered at 1, 3, 6, 12 and 24 months after transplant.
- Short Form-36 Health Survey (SF-36) [ Time Frame: 24 months ]The SF-36 will be administered at 1, 3, 6, 12 and 24 months after transplant.
- Simple Endoscopic Activity Score (SES) [ Time Frame: 24 months ]The SES will be assessed at 6, 12 and 24 months after HSCT.
- Crohn's Disease Endoscopic Index of Severity (CDEIS) [ Time Frame: 24 months ]The CDEIS will be assessed at 6, 12 and 24 months after HSCT.
- Rutgeerts endoscopic score [ Time Frame: 24 months ]Rutgeerts endoscopic score will be assessed at 6, 12 and 24 months after HSCT.
- Harvey & Bradshaw Index (HBI) [ Time Frame: 24 months ]The HBI will be assessed at 1, 3, 6, 12 and 24 months after HSCT.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03000296
|Contact: Milton A Ruiz, MD, PhDemail@example.com|
|Contact: Priscila Samara, MScfirstname.lastname@example.org|
|Sao Jose do Rio Preto, SP, Brazil, 15015-750|
|Contact: Ana Lucia, secretary +5517 33047091 email@example.com|
|Principal Investigator:||Milton A Ruiz, MD, PhD||Beneficencia Portuguesa|