SCOT Scleroderma Treatment Alternative Registry (STAR Registry)
Status:
Completed
Trial end date:
2016-03-01
Target enrollment:
Participant gender:
Summary
The Scleroderma Cyclophosphamide Or Transplant (SCOT) Trial is a Phase II/III interventional
trial comparing two treatments for early, severe scleroderma. These two interventions are
high dose immunosuppressive therapy followed by autologous stem cell transplantation and
monthly high dose pulse cyclophosphamide (the later for 12 doses). While standard of care
might be considered the optimal control arm for a trial such as this one, no such standard of
care is available for the population of scleroderma patients defined by the eligibility
criteria for this trial. The rheumatologists on the protocol team believe that the SCOT
cyclophosphamide regimen represents the best control arm for this study. However, given
concerns over use of a treatment arm as a control that has not been established as a standard
of care, this registry was established. The registry will be a prospective, observational
study of subjects with severe systemic sclerosis (SSc) who are eligible to participate in the
Scleroderma Cyclophosphamide or Transplantation (SCOT) Study but are denied insurance
coverage or decline to participate prior to randomization. Subjects will be accrued over the
same period as the SCOT study. Subjects will follow the course of treatment prescribed by
their treating physician with no interference from the registry.
The primary purpose of this study is to document the disease course and outcome in a group of
participants who are eligible for the SCOT study, but declined to participate, in order to
determine whether their outcome is better, worse, or no different than those who participate
in the treatment phase of the trial.
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)