Scleroderma Treatment With Autologous Transplant (STAT) Study
Summary
This phase II trial studies how well giving cyclophosphamide and anti-thymocyte globulin together followed by peripheral blood stem cell transplant (PBSCT) and mycophenolate mofetil works in treating patients with systemic scleroderma (SSc). Stem cells are collected from the patient's blood and stored prior to treatment. To store the stem cells patients are given colony-stimulating factors, such as filgrastim (G-CSF) or chemotherapy (cyclophosphamide) to help stem cells move from the bone marrow to the blood so they can be collected and stored. After storage, patients are then given high-dose chemotherapy, cyclophosphamide, and immunosuppression with anti-thymocyte globulin to suppress the immune system to prepare for the transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy and immunosuppression. After the stem cells have "engrafted" and have matured enough to support the immune system at approximately 2-3 months, patients are given a medication called mycophenolate mofetil (MMF) or Myfortic. This medication is given to prevent worsening or reactivation of SSc and is referred to as maintenance therapy.
Timeline
- Start
- 2011-09-15
- Primary completion
- 2023-09-15
- Completion
- 2024-09-11
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE) | Protein / enzyme biologic | — | Intravenous |
| Subject | Cyclophosphamide | Other / unclassified | — | Intravenous |
| Subject | Mycophenolate Mofetil | Small molecule | — | Oral |
| Background | Plerixafor | Small molecule | — | Subcutaneous |
| Background | filgrastim | Protein / enzyme biologic | — | Subcutaneous |