A Prospective, Observational Registry of Patients with Fabry Disease (AT1001-030)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  This is a prospective, multi centre, multinational, observational, safety and effectiveness study

Study drug and medical condition

• Name of medicine:
  GALAFOLD
• Medical condition to be studied:
  Fabry's disease

Population studied

• Age groups:
  • Paediatric Population (< 18 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)
• Special population of interest:
  Pregnant women

  Renal impaired
• Estimated number of subjects:
  450

Study design details

• Main study objective:
  This is a registry to evaluate the effects of treatment on long-term safety, effectiveness, andquality of life (QOL) in patients with Fabry disease, with a main focus on migalastat.Occurrence of key indicators of safety and effectiveness will be evaluated, such as serious adverse events, cardiac,cerebrovascular and renal events, and overall survival.
• Data analysis plan:
  Descriptive statistics will be presented. No formal hypothesis testing will be performed. Alldata will be summarized by treatment group. Continuous variables will be summarized usingthe number of observations (n), mean, standard deviation (SD), median, minimum, andmaximum. Categorical data will be summarized using counts and percents. For event data,ie, SAEs and the Fabry Associate Clinical Events (FACEs) of cardiac events, cerebrovascular events and renal events, exposure-adjusted incidence rates will be presented as counts of patients with a new event per 100 person-years exposure, with 95% confidence intervals using Ulm's method. An analysis of recurrent events will be conducted for SAEs and each specific FACE event and will be presented as total counts of each event per 100 person-years. A Cox proportional hazards ratio model will be used for the summary of survival data and will be adjusted forage at baseline and any previous cardiovascular events.