An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients with Hemophilia B (CSL222_4001)

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

  Other
• Non-interventional study design, other:
  Observational, post-authorization, long-term follow-up study

Study drug and medical condition

• Name of medicine:
  HEMGENIX
• Name of medicine, other:
  FIX prophylaxis
• Study drug International non-proprietary name (INN) or common name:
  ETRANACOGENE DEZAPARVOVEC
• Anatomical Therapeutic Chemical (ATC) code:
  (B02BD) Blood coagulation factors

  Blood coagulation factors
• Medical condition to be studied:
  Factor IX deficiency
• Additional medical condition(s):
  Hemophilia B

Population studied

• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 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 is: To investigate the short and long-term effectiveness profile of HEMGENIX by following adults with hemophilia B who are treated with HEMGENIX or are on continuous FIX prophylaxis for a period of 15 years.
• Outcomes:
  Bleeding Rate, ; Efficacy: Bleeding Rate, FIX activity, Annualized consumption of FIX replacement therapy, Number of patients remaining free of previous continuous routine prophylaxis, Target joints ; Safety: Incidence of related SAEs during 15 years of follow-up, Incidence of AESIs during 15 years of follow-up
• Data analysis plan:
  Detailed description of the statistical analyses is included in the Statistical analysis plan (SAP) document. Formal hypothesis testing is not planned for this study, and therefore no control for type I error will be done for either primary or secondary endpoint analysis. The efficacy of interest of the study is to estimate the ABR after HEMGENIX treatment (full dose or partial dose), regardless of additional FIX replacement therapy used for bleeding count. Five half-lives of FIX time will be excluded from the time at risk. Baseline demographics and characteristics will be summarized for both the HEMGENIX and FIX Prophylaxis cohorts of the study using descriptive statistics (number of observations in each group, mean, standard deviation, median, 1st quartile, 3rd quartile). No statistical comparisons are planned between the 2 cohorts. No case-matched controlling will be performed between the HEMGENIX and FIX Prophylaxis cohorts for this study.