Congenital hemophilia A (HA), Synovitis
Conditions
Brief summary
Proportion of patients with treatment response (TR) after 12 months. TR is defined as a reduction or resolution of synovial hypertrophy in at least one affected joint according to the synovial hypertrophy domain of the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) score performed by blinded ultrasound expert (BLUE) team
Detailed description
related to pharmacokinetics and efficacy for bleed prevention of ERT: ■ FVIII trough level ■ Time spent at FVIII activity >30 IU/dL ■ Time spent at FVIII activity >50 IU/dL ■ Annualized bleeding rate (ABR, all treated bleeds) and annualized joint bleeding rate (AjBR, treated joint bleeds only), related to assessments and clinical relevance of synovitis: ■ Proportion of joints with TR ■ Proportion of patients with complete resolution of synovitis in all of their joints, related to safety of efanesoctocog alfa: ■ Treatment-emergent adverse events (TEAE)
Interventions
Sponsors
GWT-Tud GmbH
Eligibility
Sex/Gender
Male
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of patients with treatment response (TR) after 12 months. TR is defined as a reduction or resolution of synovial hypertrophy in at least one affected joint according to the synovial hypertrophy domain of the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) score performed by blinded ultrasound expert (BLUE) team | — |
Secondary
| Measure | Time frame |
|---|---|
| related to pharmacokinetics and efficacy for bleed prevention of ERT: ■ FVIII trough level ■ Time spent at FVIII activity >30 IU/dL ■ Time spent at FVIII activity >50 IU/dL ■ Annualized bleeding rate (ABR, all treated bleeds) and annualized joint bleeding rate (AjBR, treated joint bleeds only), related to assessments and clinical relevance of synovitis: ■ Proportion of joints with TR ■ Proportion of patients with complete resolution of synovitis in all of their joints, related to safety of efanesoctocog alfa: ■ Treatment-emergent adverse events (TEAE) | — |
Outcome results
None listed