Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome
|ClinicalTrials.gov Identifier: NCT00731328|
Recruitment Status : Completed
First Posted : August 11, 2008
Last Update Posted : December 31, 2015
RATIONALE: Giving low doses of chemotherapy and antithymocyte globulin before a donor stem cell transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining abnormal cells (graft-versus-tumor effect).
PURPOSE: This phase II trial is studying how well a donor stem cell transplant works after busulfan, fludarabine, methylprednisolone, and antithymocyte globulin in treating patients with bone marrow failure syndrome.
|Condition or disease||Intervention/treatment||Phase|
|Leukemia Myelodysplastic Syndromes Nonmalignant Neoplasm Paroxysmal Nocturnal Hemoglobinuria||Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation||Phase 2|
- To evaluate the efficacy of HLA-haploidentical familial donor hematopoietic stem cell transplantation after reduced-intensity conditioning regimen comprising busulfan, fludarabine phosphate, and anti-thymocyte globulin in patients with bone marrow failure syndromes.
- Reduced-intensity conditioning regimen: Patients receive busulfan IV daily on days -7 and -6, fludarabine phosphate IV over 30 minutes on days -7 to -2, anti-thymocyte globulin (ATG) IV over 4 hours on days -4 to -1, and methylprednisolone IV over 30 minutes starting 30 minutes before ATG on days -4 to -1.
- HLA-haploidentical donor hematopoietic stem cell transplantation: Patients receive donor hematopoietic stem cells via Hickman catheter over 1 hour on days 0 or 1.
- Graft-versus-host-disease prophylaxis (GVHD): Patients receive cyclosporine IV over 2-4 hours every 12 hours starting on day -1 (cyclosporine can be given orally once oral medication can be tolerated) and methotrexate IV on days 2, 4 , 7, and 12. In the absence of GVHD, cyclosporine is tapered starting between days 30 to 60.
After completion of study treatment, patients are followed periodically for 1 year.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||HLA-HAPLOIDENTICAL FAMILIAL DONOR HEMATOPOIETIC CELL TRANSPLANTATION AFTER REDUCED INTENSITY CONDITIONING OF BUSULFAN, FLUDARABINE, AND ANTI-THYMOCYTE GLOBULIN FOR PATIENTS WITH BONE MARROW FAILURE SYNDROME - A PHASE 2 STUDY|
|Study Start Date :||April 2008|
|Actual Primary Completion Date :||December 2013|
|Actual Study Completion Date :||December 2013|
- Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation
infusion of mobilized donor hematopoietic progenitor cells
- Donor cell engraftment [ Time Frame: 10-35 days after transplantation ]neutrophil count over 500/ul
- Regimen-related toxicities as assessed by NCI's Common Toxicity Criteria [ Time Frame: 0-60 months after transplantation ]various toxicities of treatment
- Acute and chronic GVHD [ Time Frame: 15-100 days; 100 days to 4 years ]ocurrence of acute or chronic GVHD after transplantation
- overall survival [ Time Frame: 0-60 months ]patients surviving after transplantaion
- event-free survival [ Time Frame: 0-60 months after transplatation ]patients undergoing transplantation and maintaining donor hematopoiesis
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00731328
|Korea, Republic of|
|Asan Medical Center - University of Ulsan College of Medicine|
|Seoul, Korea, Republic of, 138-736|
|Principal Investigator:||Kyoo H. Lee, MD||Asan Medical Center|