Disease Registry for Patients with Digital Ulcers associated with Systemic Sclerosis (DUO Registry)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Drug utilisation

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  Scleroderma associated digital ulcer

Population studied

• Short description of the study population:
  Patient with Systemic sclerosis and ongoing digital ulcer disease at time of enrolment.
• Age groups:
  • Paediatric Population (< 18 years)

    • Neonate

      • Preterm newborn infants (0 – 27 days)
      • Term newborn infants (0 – 27 days)
    • Infants and toddlers (28 days – 23 months)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Estimated number of subjects:
  6108

Study design details

• Main study objective:
  For Tracleer treated patients only: to document adherence to the Summary of Product Characteristics (SmPC) requirements (liver enzymes monitoring, pregnancy testing and contraception methods) and to describe the occurrence of specific safety (e.g. pregnancy and discontinuation reasons).For ALL patients: to describe DU disease history, baseline status and disease course.
• Outcomes:
  Proportion compliance to SmPC, occurrence of new DUs, and complications and interventions associated with DUs.
• Data analysis plan:
  Safety of Tracleer: cumulative event rates, Adherence to Tracleer SmPC requirements: Proportion of patients with contraception, pregnancy testing and LFT monitoring, DU disease history, patient entry characteristics and disease course: descriptive statistics by treatment group, time to event estimate.