Scleroderma Treatment With Autologous Transplant (STAT) Study
Status:
Active, not recruiting
Trial end date:
2025-01-31
Target enrollment:
Participant gender:
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.