XTEND-Kids clinical trial1,2
A 52-week, multinational, multicenter, single-arm, open-label Phase 3 trial was conducted in previously treated patients <12 years with severe hemophilia A
* A subject in the <6 age group who had a positive inhibitor result at baseline (pre-exposure to study drug) and was withdrawn after 3 exposure days was excluded.1
† A subject in the 6 to <12 age group who received an intense consolidation treatment (2 to 3 infusions per week) for 15 weeks, after 2 traumatic hip joint bleeds was excluded from the efficacy analysis as the subject did not receive the weekly prophylaxis treatment as specified in the protocol for an extended period.1
Primary Endpoint3:
The occurrence of inhibitor development
Secondary Endpoints3:
ABR for treated bleeds by type and location
Pharmacokinetics
Perioperative management
Safety and tolerability
Treatment of bleeds
Indication
ABR=annualized bleed rate; IU=international unit.
References: 1. ALTUVIIIO Prescribing Information. Bioverativ Therapeutics Inc. Waltham, MA. 2. Data on file, May 2023. 3. Malec L, et al. Efanesoctocog Alfa Prophylaxis for Previously Treated Patients <12 Years of Age With Severe Hemophilia A. Oral presentation at: 2023 International Society on Thrombosis and Haemostasis (ISTH); June, 2023; Montreal, Canada.
