On-demand Trial Results: Overall Efficacy
Methods
COAGADEX was studied in a prospective, open-label, multicenter, non-randomized phase III study in 16 adult and adolescent subjects (≥12 years old) with severe/moderate factor X deficiency (Study TEN01)
- 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
- Baseline 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
Results
In this study, 87.3% of non-menorrhagic bleeds were covert
- In this study, 16 subjects experienced a total of 187 bleeds that were assessed by subjects and an independent data review committee consisting of 3 hemophilia experts
| Types of Bleeds that Occurred in the Trial | n (%) of bleeds N=187 |
|---|---|
| Bleed type | |
| Covert | 110 (58.8) |
| Menorrhagic | 61 (32.6) |
| Overt | 16 (8.6) |
| Bleed location | |
| Mucosal | 73 (39.0) |
| Joint | 63 (33.7) |
| Muscle | 26 (13.9) |
| Other* | 25 (13.4) |
| Bleed cause | |
| Spontaneous | 79 (42.2) |
| Menorrhagia | 61 (32.6) |
| Injury | 47 (25.1) |
| Bleed severity† | |
| Major | 98 (52.4) |
| Minor | 88 (47.1) |
| Not evaluated | 1 (0.5) |
*Includes cut/wound, concussion, foot, forehead, hand, kidney, left forearm, lumbar area, right shoulder, subcutaneous and thumb.
†As categorized by the data review committee.
-
98% of bleeds were controlled with 1 or 2 infusions of COAGADEX
Infusions of COAGADEX Needed to Control Bleeding
- Mean (SD) dose per infusion was 25.3 (2.4) IU/kg
-
98.4% of bleeds were treated successfully (defined as “excellent” or “good response”)
Subject Assessment of Treatment Response with COAGADEX
Rating Scale for Efficacy of COAGADEX
| Efficacy rating | Overt bleeds | Covert bleeds | Menorrhagic bleeds |
|---|---|---|---|
| Excellent | Bleeding stopped within 12h after dosing with COAGADEX, with only 1 dose required | 1 dose of COAGADEX was required or 2 doses of COAGADEX were required less than 48h apart | 1 dose of COAGADEX was required or 2 doses of COAGADEX were required less than 48h apart |
| Good | Bleeding stopped within 24h after the first dose of COAGADEX, with 1 or 2 doses required | 3 doses of COAGADEX were required, with less than 48h between the first and last dose | 2 doses of COAGADEX were required, with more than 48h between the first and the last dose |
Reference: Austin S, Kavakli K, Norton M, et al. Haemophilia. 2016;22:419-425.