SCOT Scleroderma Treatment Alternative Registry (STAR Registry)
Condition: Scleroderma, Systemic; Sclerosis
MeSH terms: Scleroderma, Systemic; Scleroderma, Diffuse; Scleroderma, Localized
Study type: Observational

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.
    

Detailed description: 
      For multiple reasons, the SCOT investigators and the sponsor of the SCOT trial, the Division
      of Allergy, Immunology, and Transplantation (DAIT) of the National Institute of Allergy and
      Infectious Diseases (NIAID), determined that it is important to track the course of a
      'matched' group of patients, who are not exposed to these treatments but receive currently
      available therapy in the community. First, such a group will provide information to determine
      if the SCOT entry criteria do indeed identify these high-risk individuals. More importantly,
      such a group of patients is likely to be treated with a variety of medical regimens,
      including some immunosuppressive therapy with cyclophosphamide or other immunosuppressive
      agents that may modify the natural history of the disease. In evaluating the relative
      efficacy of the two treatment regimens, it will be important to assess whether outcomes in
      the subjects treated under the SCOT protocol have outcome profiles that differ from those
      associated with the matched group of patients treated in the community. One readily available
      group that meets these criteria are those individuals who are otherwise eligible for the SCOT
      trial but fail to be randomized because they either decline to participate or are denied
      insurance coverage to receive the SCOT treatment regimens.

      The duration of this trial is 44 months. Participants will be enrolled over the same period
      as the SCOT trial. Participants will follow the course of treatment prescribed by their
      treating physician with no interference from the registry. All participant contact, including
      obtainment of informed consent and telephone interview regarding outcome measurements will be
      performed by SCOT study personnel at the University of Texas, Houston (one of the SCOT
      transplant centers). Participants will be contacted by phone every 3 months to determine
      vital status, record medical and other therapy, and administer the modified Scleroderma
      Health Assessment Questionnaire (S-HAQ). Medical records will be obtained to verify
      self-reported medical events.
    

Intervention name(s): currently available therapy in the community
Intervention type(s): Drug
Intervention description(s): Participants will receive telephone calls every 3 months for approximately 44 months for the purpose of outcome surveys
Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Participants with severe systemic sclerosis who are eligible for the SCOT study, but either
        decline to participate or are denied insurance coverage, prior to randomization will be
        invited to participate in this registry.