A Post-Authorization, Multicenter, Multinational, Longitudinal, Observational Safety Registry Study for Patients Treated with Voretigene Neparvovec

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

• Name of medicine:
  Luxturna
• Study drug International non-proprietary name (INN) or common name:
  VORETIGENE NEPARVOVEC
• Anatomical Therapeutic Chemical (ATC) code:
  (S01XA27) voretigene neparvovec
• Medical condition to be studied:
  Hereditary retinal dystrophy

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  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:
  40

Study design details

• Main study objective:
  The objective of this post-authorization observational study is to collect long-term safety information (i.e. for 5 years after treatment) associated with voretigene neparvovec (vector and/or transgene), its subretinal injection procedure, the concomitant use of corticosteroids, or a combination of these procedures and products.
• Outcomes:
  Frequency of adverse events, serious adverse events and adverse events of special interest, Pregnancy outcomes, and visual function measures.
• Data analysis plan:
  This study is intended to descriptively document the frequency and severity of events of interest related to voretigene neparvovec (vector and/or transgene), the subretinal injection procedure, the concomitant use of corticosteroids, or a combination of these procedures and products. The Full Analysis Set (FAS), all enrolled individuals with a signed informed consent/assent, will be used to summarize all data.Continuous variables will be summarized in terms of mean, standard deviation, median, minimum and maximum (and other descriptive statistics when appropriate). Categorical variables will be summarized using frequency counts and percentages. Unless otherwise noted, the denominator for the percentages will be the FAS population.Demographics and pretreatment/baseline disease characteristics will be summarized. Ophthalmic examination and patient/caregiver questionnaire results will be presented at pre-treatment/baseline and post-administration/annually.