Pilot Study of Stem Cell Transplantation for Children and Young Adults With Refractory Crohn's Disease.
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|ClinicalTrials.gov Identifier: NCT02225795|
Recruitment Status : Withdrawn (recruitment difficulty)
First Posted : August 26, 2014
Last Update Posted : February 7, 2018
This is a study for people with Crohn's Disease (CD) that are not responsive to standard treatment. CD is a chronic disease with an auto-immune component that goes away and relapses over the years and causes lifelong impairment of health and quality of life. Regardless of the therapy used some patients remain seriously ill with active disease after multiple therapeutic options have been exhausted. There is currently no drug that will cure CD. Drug treatment is focused on controlling symptoms. Another treatment is to perform surgery but again this does not lead to cure and is often linked to infection, short gut syndrome problems and psycho-social and cosmetic issues. Therefore, a treatment that does not involve surgery or long-term drug treatment may be beneficial especially to young adults.
Hematopoietic stem cell transplantation (HSCT) has been of value in other auto-immune diseases and it is possible that it could be of value in CD. This is a pilot study to determine if HSCT is safe and effective for children and young adults with severe CD. For this study the stem cells will come from the patient. This is called an autologous transplant. The patient will undergo collection and storage of his/her peripheral blood stem cells (PBSC). The patient will be given drugs to move (or mobilize) the stem cells from his/her bone marrow into his/her blood where they will be collected on a machine called apheresis (similar to dialysis.) The cells will be stored and given back to the patient about 1 month after collection.
|Condition or disease||Intervention/treatment||Phase|
|Crohn's Disease||Other: Hematopoietic stem cell transplantation||Not Applicable|
Show Detailed Description
|Study Type :||Interventional|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Study of Autologous Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide for Children and Young Adults With Refractory Crohn's Disease.|
|Study Start Date :||August 2014|
|Estimated Primary Completion Date :||August 2019|
|Estimated Study Completion Date :||December 2019|
Experimental: Single Arm: All subjects
Hematopoietic stem cell transplantation
Other: Hematopoietic stem cell transplantation
Immuno-ablative regimen for HSCT:
- To determine the feasibility and toxicity of immunoablation with HSCT and post-HSCT infusion of cyclophosphamide in children and young adults with refractory Crohn's disease (CD). [ Time Frame: first 100 days post HSCT ]This is a composite outcome to determine the feasibility and toxicity of immunoablation with HSCT and post-HSCT infusion of cyclophosphamide in children and young adults with refractory Crohn's disease (CD). Death (transplant related mortality, TRM) and severe non-hematologic toxicity (≥ grade 3 toxicity; NCI Toxicity Criteria version 4.0) within the first 100 days after HSCT will be monitored to meet this end-point.
- 1. Incidence of viral reactivations - CMV and EBV [ Time Frame: First 100 days post HSCT ]
- 2. Incidence of invasive fungal infections [ Time Frame: within first 100 days post HSCT ]
- 3. Immune reconstitution after HSCT [ Time Frame: Until the study is closed which is anticipated to be approximately 3 years. ]3. Immune reconstitution after HSCT lymphocyte subpopulations (absolute number of CD3, CD4, CD8 and CD19 cells), immunoglobulin levels (IgG) and CD4+ CD25+ CD127+ regulatory T cells (Treg) populations on day +30 , +60 , +100, day +180 and yearly post-HSCT.
- 4. Evaluate the efficacy and benefit of HSCT in this population at 1 year post HSCT, by serial assessments of clinical activity of CD, assessment of disease severity- CDAI scores and length of steroid free remissions. [ Time Frame: 1 year post transplant ]
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): NCT02225795
|Principal Investigator:||Haydar Frangoul, MD||TriStar Health|