World Federation of Hemophilia Gene Therapy Registry (WFH GTR)

Study type

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

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  Haemophilia

Population studied

• Age groups:
  • 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:
  500

Study design details

• Main study objective:
  The primary objective of the WFH GTR is to determine the long-term safety of factor VIII and factor IX gene therapies in patients with hemophilia. The secondary objectives of the WFH GTR are to determine the long-term efficacy and the durability of factor VIII and factor IX gene therapies in patients with hemophilia and to assess long-term quality of life.
• Outcomes:
  The primary outcome/endpoint is safety events over the long-term. The secondary outcomes/endpoints are efficacy and durability of efficacy of factor VIII and factor IX gene therapies in patients with hemophilia
• Data analysis plan:
  The WFH GTR study is a prospective, observational, and longitudinal registry of patients diagnosed with hemophilia, who have received gene therapy for hemophilia. The primary objective is to determine the long-term safety of factor VIII and factor IX gene therapies. Safety will be analyzed from adverse events (AE) and mortality using Kaplan-Meier plots. The secondary objectives are to determine the long-term efficacy and the durability of factor VIII and factor IX gene therapies. Bleeding rate and plasma factor activity level will be assessed together with long-term quality of life. Bleeding event rate will be calculated as events by patient-year(s) as number of incident cases divided by the amount of person-time at risk. Median time to occurrence of each bleed will be calculated using Kaplan-Meier plots. For long-term quality of life, changes in the composite health related quality of life scores will be reported as mean, median, standard deviation and interquartile range.