On-demand Trial Results: Efficacy
COAGADEX was studied in a prospective, open-label, multicenter, non-randomized phase III study in 16 patients with severe/moderate factor X deficiency (Study Ten 01)1,2
- COAGADEX was administered on demand or for short-term prophylaxis for 6 months to 2 years until ≥12 bleeding episodes had been treated with the product
- Key inclusion criteria:
- Aged ≥12 years
- Basal plasma factor X concentration <5 IU/dL
- Factor X replacement therapy for ≥1 spontaneous or menorrhagic bleeds in the past year
- Key exclusion criteria:
- History of factor X inhibitor development
- Positive for factor X inhibitors at the screening visit
- Known thrombocytopenia or clinically significant renal or liver disease
*Includes cut/wound, central nervous system, epistaxis, forehead, gastrointestinal, intracranial, intraperitoneal, mouth, mucosal (not menorrhagia), pelvic, rectal, and unknown.
†As categorized by the data review committee.
In this study, 16 patients experienced a total of 187 bleeds that were assessed by patients and an independent data review committee consisting of 3 hemophilia experts
98% of bleeds were controlled with 1 or 2 infusions of COAGADEX
- Mean dose per infusion was 25.3 IU/kg
- Mean total dose per bleed was 30.4 IU/kg
98.4% of bleeds were treated successfully (defined as “excellent” or “good”)
Reference: 1. Austin S, Norton M, et al. Safety and efficacy of FACTOR X, a new high-purity factor X concentrate: a phase 3 study in patients with hereditary factor X deficiency. Poster presented at the 9th Annual Scientific Symposium of the Hemostasis and Thrombosis Research Society (HTRS), New Orleans, April 16-18 2015. 2. COAGADEX® (Coagulation Factor X, Human) Prescribing Information. Durham, NC: BPL Limited. 2015.