T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders
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|ClinicalTrials.gov Identifier: NCT00176826|
Recruitment Status : Terminated (Replaced by another protocol)
First Posted : September 15, 2005
Results First Posted : April 28, 2014
Last Update Posted : January 23, 2018
|Condition or disease||Intervention/treatment||Phase|
|Hemophagocytic Lymphohistiocytosis X-Linked Lymphoproliferative Disorders Chediak-Higashi Syndrome Griscelli Syndrome Immunologic Diseases Langerhans-Cell Histiocytosis Hematologic Diseases||Procedure: Stem Cell Transplant Drug: Myeloablative conditioning regimen||Phase 2 Phase 3|
Subjects will begin chemotherapy as a preparative regimen, which is intended to completely eliminate their defective immune system and bone marrow. The preparative regimen consists of the chemotherapy drugs (busulfan, cyclophosphamide, and antithymocyte globulin (ATG)).
Transplantation: subjects will then have a source of blood stem cells (bone marrow) from their donor administered into their catheter. Medication will be given to help prevent Graft-Versus Host Disease (GVHD). The ATG will help to deplete the donor stem cells of the type of cells that can cause GVHD and will also help to promote engraftment of the new stem cells.
Recovery Phase: The second phase of treatment consists of a period after transplantation during which we wait for the return of bone marrow function. This usually takes two to four weeks. Subjects will be given a blood cell growth factor, G-CSF, to help speed recovery of the white blood cells and potentially decrease the risk of infection and decrease the time until the bone marrow recovers.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||22 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||In-vivo T-cell Depletion and Hematopoietic Stem Cell Transplantation for Life-Threatening Immune Deficiencies and Histiocytic Disorders|
|Study Start Date :||September 2000|
|Actual Primary Completion Date :||August 2012|
|Actual Study Completion Date :||August 2015|
Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
Procedure: Stem Cell Transplant
Infusion of hematopoietic stem cells (bone marrow, cord blood, peripheral blood stem cells) following myeloablative conditioning regimen.
Other Name: HSCTDrug: Myeloablative conditioning regimen
Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
- Time to Transplant Engraftment [ Time Frame: Day 100 Post Transplant ]
- Number of Patients With Treatment Related Mortality. [ Time Frame: Day 100 Post Transplant ]
- Number of Patients Surviving (Disease-free) [ Time Frame: 1 year ]
- Number of Patients With Grade II-IV Graft-Versus-Host Disease (GVHD) [ Time Frame: Day 100 Post Transplant ]
- Number of Patients With Graft Failure [ Time Frame: Day 100 Post transplant ]
- Number of Patients With III-IV Graft-Versus-Host Disease (GVHD) [ Time Frame: Day 100 Post Transplant ]
- Number of Patients Surviving (Disease-free) [ Time Frame: 3 years ]
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): NCT00176826
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota|
|Minneapolis, Minnesota, United States, 55455|
|Principal Investigator:||Angela Smith, MD||University of Minnesota Medical Center|