C0371017 - A PHASE 3, NON-INVESTIGATIONAL PRODUCT, MULTI COUNTRY COHORT STUDY TO DESCRIBE THE LONG-TERM SAFETY AND EFFECTIVENESS OF A PRIOR SINGLE-DOSE TREATMENT WITH INVESTIGATIVE GIROCTOCOGENE FITELPARVOVEC OR FIDANACOGENE ELAPARVOVEC IN PARTICIPANTS WITH HEMOPHILIA A OR HEMOPHILIA B, RESPECTIVELY

Condition studied

severe to moderately severe hemophilia B (FIX:C ≤ 2%), hemophilia A

Investigational medicinal product(s)

Eligibility

Primary endpoint

Incidence of thromboembolic events, Incidence of factor inhibitor development, Incidence of hepatic malignancy, Incidence of liver abnormalities, Factor activity level

Endpoint detail

Total ABR (treated and untreated), Incidence of and time from previously administered gene therapy infusion to resumption of prophylaxis, AIR of exogenous factor, Consumption of exogenous factor, Incidence of Non-hepatic malignancy, Incidence of Auto-immune disorders, Incidence of SAEs, All cause-mortality, EQ-5D-5L dimension and VAS scores