Study type

Study type

Non-interventional study

Scope of the study

Effectiveness study (incl. comparative)
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Name of medicine

ROCTAVIAN

Medical condition to be studied

Factor VIII deficiency
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

200
Study design details

Main study objective

To describe the bleeding profile and long-term durability of coagulation factor VIII (FVIII) expression in subjects administered with ROCTAVIAN™

Outcomes

Annualized Bleeding Rate (treated and all bleeds) Percentage of subjects with zero (0) bleeds (treated and all bleeds), by treatment duration Change over time in FVIII activity levels Use of exogenous factor and non-factor replacement treatment Change in clinical outcome assessments over time. Proportion, event rate, and incidence rate of SAEs. Proportion, event rate, and incidence rate of suspected ADRs Proportion, event rate, and incidence rate of TAEs of hepatotoxicity, thromboembolic events, infusion reactions (including hypersensitivity), new malignancies, and development of FVIII inhibitors

Data analysis plan

Due to the observational nature of this study and inclusion criteria, results will represent a broad range of real-world practice, which may reflect or be influenced by site heterogeneity (e.g. clinic structure, local clinical practice) ), differences in clinical practice and underlying differences in the patient population. Consistent and thorough data collection should allow results to be described in such a way that these differences can be characterized, analyzed, and reported. Full analysis will be described in the SAP.