Study of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) for On-demand Treatment of Bleeding Episodes in Patients With Hemophilia A or B With Inhibitors
Hemophilia A With Inhibitors, Hemophilia B With Inhibitors
About this trial
This is an interventional treatment trial for Hemophilia A With Inhibitors
Eligibility Criteria
Inclusion Criteria:
- Male subjects with hemophilia A or B and inhibitors.
- Age ≥ 12 and ≤ 65 years.
- High responding inhibitor with documented historical inhibitor titer > 5 Bethesda Units/mL.
Exclusion Criteria:
- Congenital or acquired coagulation disorders other than hemophilia A or B.
- Ongoing immune tolerance induction therapy or planned during study.
- Known or suspected hypersensitivity to activated recombinant human FVII or to any excipient of CSL689.
- Body mass index > 30 kg/m².
- Major surgery within 28 days before screening or scheduled major and / or orthopedic surgery during the study.
- Advanced atherosclerotic disease (ie, known history of ischemic heart disease, or ischemic stroke).
- Any clinical signs or known history of thromboembolic events, including known deep vein thrombosis.
- Human immunodeficiency virus (HIV)-positive subjects who have low cluster of differentiation 4 (CD4)+ lymphocyte count (200/μL or less) at screening.
- Use of the following within the screening period or planned during study: a) plasma or coagulation factor concentrates other than rescue therapy or therapy during Part 1, b) other platelet inhibitors, c) desmopressin, and d) fibrinolysis inhibitors, except if used as local treatment (eg, for oral bleeds).
Sites / Locations
- Site Reference # 2680001
- Site Reference # 3800023
- Site Reference # 4580001
- Site Reference # 6430026
- Site Reference # 7100001
- Site Reference # 7240007
- Site Reference # 7640006
- Site Reference # 7640004
- Site Reference # 8040005
- Site Reference # 8260008
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Experimental
Experimental
Active Comparator
Active Comparator
CSL689 low-dose
CSL689 high-dose
Eptacog alfa low-dose
Eptacog alfa high-dose
Part 1: single injection of low-dose CSL689 for PK evaluation Part 2: up to 2 injections of low-dose CSL689 per bleeding event (bleeding events 1 to 3*) Part 3: up to 3 injections of low-dose CSL689 per bleeding event * Note: All subjects in the low-dose arm will be treated with high-dose CSL689 for bleeding events 4-6 in Part 2
Part 1: single injection of high-dose CSL689 for PK evaluation Part 2: up to 2 injections of high-dose CSL689 per bleeding event (bleeding events 4 to 6*) Part 3: up to 3 injections of high-dose CSL689 per bleeding event Note: All subjects in the high-dose arm will be treated with low-dose CSL689 for bleeding events 1-3 in Part 2
Single injection of low-dose Eptacog alfa in Part 1 for PK evaluation
Single injection of high-dose Eptacog alfa in Part 1 for PK evaluation