For Interested Participants
Are You Interested in Participating in the SCOT Study?
More clinical research studies are needed to determine the best treatment for scleroderma. While organ-specific treatment is important and helpful, some experts believe that a broader and possibly more effective approach might be to treat the individual’s immune system as a whole. SCOT study investigators will study two immune-based approaches:
- Stem Cell Transplant: High-dose immunosupression followed by stem cell transplantation
- High-Dose Cyclophosphamide (Cytoxan): Monthly intravenous Cytoxan for 1 year
Why Were These Approaches Chosen?
The initial idea for using stem cell transplantation comes from animal experiments, as well as observations from cancer patients who also had an autoimmune disease that improved after undergoing bone marrow transplantation. In most cases, their autoimmune disease went into long-term remission after the transplant. For the SCOT study, only “autologous” (self) stem cell transplantation will be used. High-dose immunosuppressive therapy is given and participants then receive back their own purified blood stem cells to restore their immune system.
High-dose Cytoxan is being studied because it has strong immunosuppressive properties. It reduces the numbers of immune cells thought to be causing scleroderma. Cytoxan has been used widely by doctors to treat autoimmune diseases, such as scleroderma and lupus. However, the dose being used in the SCOT study is higher than what doctors typically prescribe, and the length of administration is longer. The decision to test this dose is based on a variety of reports that have suggested that a higher dose may stabilize or improve symptoms of the disease. Specifically, it has been reported to slow down damage in the lungs caused by scleroderma.
Why Participate in the SCOT Study?
Participating in the SCOT study may provide you with access to an investigational approach for treating your scleroderma that you may not otherwise be able to receive. Participation in SCOT is an individual decision that should occur only after you fully understand the study and what is expected from you as a participant. There may be no direct benefit to you from participating in this study. The 2 investigational approaches being studied in SCOT are intensive and can result in death. All persons interested in this study should carefully review the possible risks and side effects described in the consent form. A research consent form may be obtained from a SCOT study center.
The SCOT Study
The SCOT study involves a screening phase to make sure you are eligible for the study. After the screening phase, if you are eligible and decide to participate, you will enter the study-procedure phase where you will be randomly assigned to the stem cell transplant or high-dose monthly Cytoxan group.
Screening Phase
The screening process may last up to 2 months and is designed to check the extent of your disease condition and determine if you are eligible for the study. You will have a variety of tests performed to find out how scleroderma has affected your internal organs. These tests are outlined in the consent form.
Study-Procedure Phase
Depending on the group to which you have been assigned, you will either undergo a stem cell transplant or receive high-dose monthly Cytoxan. At 6, 12, 24, 36, 42, 48, and 60 months after enrollment, all study participants will have a thorough evaluation to check out the function of the heart, lungs, and gastrointestinal system and the response to the study procedure. In addition, all participants will undergo frequent physical examinations and laboratory tests over the first year. After the first year, follow-up visits will occur at least every year through year 5.