Multicenter Transplant Study for Fanconi Anemia
The trial proposed is a multicenter treatment protocol designed to examine transplant related events in patients with Fanconi anemia who lack matched sib donors have severe aplastic anemia (SAA), or myelodysplastic syndrome(MDS) or acute myelogenous leukemia (AML).
Biological: Miltenyi CliniMACS
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine|
- The primary objectives are: The incidence and quality of engraftment and hematopoietic reconstitution. The incidence of early transplant related mortality The incidence and severity of acute GvHD (graft versus host disease) and chronic GvHD. [ Time Frame: 1-5 years ] [ Designated as safety issue: Yes ]
- The incidence of overall survival and disease free survival over time. [ Time Frame: 5 Years ] [ Designated as safety issue: No ]
|Study Start Date:||October 2009|
|Estimated Study Completion Date:||October 2020|
|Estimated Primary Completion Date:||October 2015 (Final data collection date for primary outcome measure)|
Experimental: Chemotherapy and Miltenyi, CliniMACS
Busulfan 0.6-1.0 mg/Kg/dose IV Q 12 hours X4 Doses Fludarabine 35 mg/m2/dose IV once daily X4 Doses Cyclophosphamide 10 mg/Kg/dose IV once daily X4 Doses Anti-thymocyte globulin - Thymoglobulin 2.5 mg/Kg/dose IV daily X4 Doses Miltenyi CliniMACS
Busulfan 0.4-1.0 mg/Kg/dose IV Q 12 hours X4 Doses Fludarabine 35 mg/m2/dose IV once daily X4 Doses Cyclophosphamide 10 mg/Kg/dose IV once daily X4 Doses Anti-thymocyte globulin - Thymoglobulin 2.5 mg/Kg/dose IV daily X4 DosesBiological: Miltenyi CliniMACS
The CliniMACs device (Miltenyi Biotec, Auburn, CA) will be employed for the CD34 selection procedure. It consists of tubing, bags, and a pair of columns, placed at appropriate locations in the Tubing Set to facilitate the cell selection process.
The trial proposed is a single arm phase II multicenter treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine and ATG (Anti-thymocyte globulin)for the treatment of patients with Fanconi anemia who have severe aplastic anemia (SAA), or myelodysplastic syndrome(MDS) or acute myelogenous leukemia (AML), lacking HLA-genotypically (HLA = human leukocyte antigen) identical donors using stem cell transplants derived from HLA-compatible unrelated donors or HLA haplotype-mismatched related donors using Miltenyi's CliniMACS.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01082133
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|Principal Investigator:||Stella Davies, MD||CCHMC|