Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders
OBJECTIVES: I. Determine the safety and long term complications of total body irradiation in combination with cyclophosphamide, anti-thymocyte globulin, and autologous CD34-selected peripheral blood stem cell (PBSC) transplantation in children with refractory autoimmune disorders.
II. Determine the efficacy of this treatment regimen in these patients. III. Determine the reconstitution of immunity after autologous CD34-selected PBSC transplantation in these patients.
IV. Determine engraftment of autologous CD34-selected PBSC in these patients.
|Systemic Sclerosis Systemic Lupus Erythematosus Dermatomyositis Juvenile Rheumatoid Arthritis Autoimmune Diseases||Procedure: Stem Cell Transplantation Device: CD34 selection||Phase 1|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||A Pilot Study of High-Dose Immunosuppression Followed by Infusion of CD34-Selected Autologous or Syngeneic Peripheral Blood Stem Cells for Treatment of Refractory Autoimmune Disorders|
- Mortality [ Time Frame: Annually for 5 years and then every 5 years thereafter ]
- Immune reconstitution, engraftment, efficacy, late-effects [ Time Frame: Annually for 5 years and then every 5 years thereafter ]
|Study Start Date:||November 2000|
|Study Completion Date:||May 2011|
|Primary Completion Date:||July 2010 (Final data collection date for primary outcome measure)|
Participants will receive a stem cell transplant.
Procedure: Stem Cell Transplantation
Participants will receive a stem cell transplantation along with irradiation and the drugs anti-thymocyte globulin, cyclophosphamide, and filgrastim as noted in the text of this record.Device: CD34 selection
CD34+ cells are separated from the rest of the peripheral blood stem cells.
PROTOCOL OUTLINE: This is a multicenter study. Patients receive filgrastim (G-CSF) subcutaneously daily until peripheral blood stem cell (PBSC) collection is completed. CD34+ cells are separated from the rest of the PBSCs.
Patients undergo total body irradiation twice daily on days -5 and -4. Patients receive anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5 and cyclophosphamide IV on days -3 and -2. CD34-selected PBSCs are reinfused on day 0. Patients receive G-CSF IV daily beginning on day 0 and continuing until blood counts recover.
Patients are followed annually for 5 years and then every 5 years thereafter.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00010335
|United States, Washington|
|Fred Hutchinson Cancer Research Center|
|Seattle, Washington, United States, 98109|
|Study Chair:||Ann Woolfrey||Fred Hutchinson Cancer Research Center|
|Principal Investigator:||Carol A. Wallace, MD||Fred Hutchinson Cancer Research Center|