Hemophilia A
Conditions
Brief summary
Occurrence of adverse drug reactions, serious adverse events, and events of special interest, including: a) Hepatotoxicity; b) Thromboembolic events; c) Development of FVIII inhibitors; d) Transmission to third parties; e) Integration with theoretical risk of tumorigenesis
Detailed description
Changes in annualized bleeding rate (ABR) (treated bleeds and all bleeds), FVIII activity measured over time (CSA and OSA), Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate), Changes in Haemo-QoL-A
Interventions
DRUGROCTAVIAN 2 × 1013 vector genomes/mL solution for infusion
Sponsors
Biomarin Pharmaceutical Inc.
Eligibility
Sex/Gender
Male
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Occurrence of adverse drug reactions, serious adverse events, and events of special interest, including: a) Hepatotoxicity; b) Thromboembolic events; c) Development of FVIII inhibitors; d) Transmission to third parties; e) Integration with theoretical risk of tumorigenesis | — |
Secondary
| Measure | Time frame |
|---|---|
| Changes in annualized bleeding rate (ABR) (treated bleeds and all bleeds), FVIII activity measured over time (CSA and OSA), Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate), Changes in Haemo-QoL-A | — |
Countries
Belgium, France, Germany, Italy, Spain
Outcome results
None listed