Conditions: Systemic Sclerosis Scleroderma
Keywords: Autologous Stem Cell Transplantation Hematopoietic Stem Cell Transplant
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 9
Healthy volunteers: no

Brief summary:
This study is designed to treat systemic sclerosis (scleroderma) patients with an autologous stem cell transplant using a regimen of immune suppressant drugs and chemotherapy that is less toxic to your heart.

Detailed description:
The autologous hematopoietic stem cell transplant used in this research study is an investigational procedure that uses cyclophosphamide and fludarabine (chemotherapy), rabbit anti-thymocyte globulin (rATG) (a protein that kills the immune cells that are thought to be causing your disease), and rituximab (a biologic drug that targets B cells of your immune system). After use of these treatments, the patient will receive their own previously collected blood stem cells (autologous stem cell transplant). The ability of these experimental treatments to stop relapses and progression (worsening) of your systemic sclerosis will be assessed.

Arms:
arm 1: Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with rituximab, fludarabine, cyclophosphamide, Mesna, rATG (rabbit), and methylprednisolone. Granulocyte-colony stimulating factor (G-CSF) and intravenous immunoglobulin (IVIg) will be administered post-transplant.

Interventions: intervention 1: Rituximab intervention 2: Fludarabine intervention 3: Cyclophosphamide intervention 4: Mesna intervention 5: rATG intervention 6: Methylprednisolone intervention 7: G-CSF intervention 8: IVIg intervention 9: Autologous Stem Cells
Intervention descriptions: intervention 1: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer intervention 2: A chemotherapy medication commonly used in the treatment of leukemia and lymphoma intervention 3: A medication used as chemotherapy and to suppress the immune system intervention 4: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder intervention 5: A rabbit polyclonal antibody to lymphocytes intervention 6: A corticosteroid medication used to suppress the immune system and decrease inflammation intervention 7: A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream intervention 8: Pooled immunoglobulin (IgG) from thousands of plasma donors that has immunomodulatory and anti-inflammatory effects intervention 9: Infusion of patient's own stem cells

Protocol document:
Prot_002.pdf: 





































































































SAP_000.pdf: