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
Phase 3 – large-scale trial before approval
Conditions:
severe to moderately severe hemophilia B (FIX:C ≤ 2%)
hemophilia A
Sponsor: Pfizer Inc.
trial.available_in:
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Overview
Trial status: Authorised
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
Incidence of thromboembolic events, Incidence of factor inhibitor development, Incidence of hepatic malignancy, Incidence of liver abnormalities, Factor activity level
Description
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
Who can participate
Age group: 18-64 years
Gender: Male
Target enrollment: 7 participants
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