Long-Term Observational study of Translarna Safety and Effectiveness in Usual Care (STRIDE)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Long-term observational registry

Study drug and medical condition

• Name of medicine:
  TRANSLARNA
• Medical condition to be studied:
  Duchenne muscular dystrophy gene carrier
• Additional medical condition(s):
  Nonsense mutation in the dystrophin gene

Population studied

• Age groups:
  • Paediatric Population (< 18 years)

    • 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:
  360

Study design details

• Main study objective:
  The study is designed to evaluate the long-term safety and effectiveness, and utilization pattern of Translarna in real-world routine clinical practice.
• Outcomes:
  The objectives of the study are: • Obtain additional information on all safety concerns being tracked in the Risk • Management Plan and the long-term safety profile of Translarna • Obtain additional information on the long-term effectiveness of Translarna. • Monitor the utilization pattern of Translarna in usual care.
• Data analysis plan:
  For any given safety event, the unadjusted incidence rate and exposure-adjusted event rate will be calculated, along with exact binomial confidence intervals. Further details of the analyses will be included in the SAP. Specific analyses on safety concerns with ataluren treatment will be performed when data available. The effectiveness of treatment with Translarna will be evaluated in the context of data were available: 6MWT, timed function tests, NSAA, PUL, cardiac and pulmonary functions. Data from these assessments will be summarized by visit. Changes in the relevant variable from baseline to each post-baseline visit will be summarized descriptively. Changes from baseline to each post-baseline visit will be analysed using paired t-tests. Results will be presented combined and stratified as appropriate. Subgroup analyses will be performed.