Hemophilia B MedDRA version: 20.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Idelvion®
Product Name: Recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP)
Product Code: rIX-FP or CSL654
Pharmaceutical Form: Powder and solvent for solution f
Sponsors
CSL Behring GmbH
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: • Male Chinese subjects aged = 70 years • Subjects with documented severe or moderately severe hemophilia B (FIX activity of = 2%) • Subjects have received FIX products for = 150 exposure days (EDs) (subjects aged = 6 years) or = 50 EDs (subjects aged =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Known hypersensitivity (allergic reaction or anaphylaxis) to any FIX product or hamster protein. • Known congenital or acquired coagulation disorder other than congenital FIX deficiency. • Currently receiving intravenous (IV) immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment. • Currently receiving a long-acting recombinant FIX treatment such as coagulation factor IX (recombinant), Fc fusion protein (Alprolix®). • Use of traditional or herbal Chinese medicine(s) with an impact on hemophilia, including coagulation, within 28 days before Day 1 and / or refusal to abstain from these during the study until the end of the subject’s participation in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the PK of 50 IU/kg rIX-FP in Chinese PTPs with hemophilia B (FIX activity of = 2%) during the Single-dose PK Period, to evaluate the efficacy of routine prophylaxis dosing with rIX-FP in preventing spontaneous bleeding episodes in Chinese PTPs with hemophilia B (FIX activity of = 2%), and to evaluate the safety of routine prophylaxis dosing with rIX-FP in Chinese PTPs with hemophilia B (FIX activity of = 2%) with respect to the development of inhibitors to FIX.;Secondary Objective: To further evaluate the PK of 50 IU/kg rIX-FP in Chinese PTPs with hemophilia B (FIX activity of = 2%) during the Single-dose PK Period, to evaluate the PK of 50 IU/kg rIX-FP in Chinese PTPs with hemophilia B (FIX activity of = 2%) during the Repeat PK Period, to further evaluate the efficacy of rIX-FP in Chinese PTPs with hemophilia B (FIX activity of = 2%), and to further evaluate the safety of rIX-FP in Chinese PTPs with hemophilia B (FIX activity of = 2%).;Primary end point(s): 1. Incremental recovery (IR) (plasma FIX activity) 2. Maximum plasma concentration (Cmax) 3. Terminal elimination half-life (t1/2) of rIX-FP 4. Area under the concentration-time curve (AUC) 5. Clearance (Cl) of rIX-FP 6. Annualized spontaneous bleeding rate (AsBR) 7. Number of subjects who develop an inhibitor to FIX;Timepoint(s) of evaluation of this end point: 1. Before, and at 30 minutes after the end of, rIX-FP infusion on Day 1 2, 3, 4, and 5. Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 6. Up to 18 months 7. Up to 18 months after rIX-FP infusion | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Percentage of area under the concentration-time curve extrapolated (%AUCExt) 2. Area under the first moment versus time curve extrapolated to infinity (AUMC0-8) 3. Mean residence time (MRT) 4. Apparent volume of distribution during the terminal phase (Vz) 5. Apparent volume of distribution at steady-state (Vss) 6. Time to reach Cmax (Tmax) 7. Elimination rate constant (?z) 8. Incremental recovery (IR) (plasma FIX activity) - Repeat PK 9. Maximum plasma concentration (Cmax) - Repeat PK 10. Terminal elimination half-life (t1/2) of rIX-FP - Repeat PK 11. Area under the concentration-time curve (AUC) - Repeat PK 12. Clearance (Cl) of rIX-FP - Repeat PK 13. Annualized bleeding rate (ABR) 14. Annualized joint bleeding rate (AjBR) 15. Clinical evaluation of hemostatic efficacy for major bleeding episodes 16. Change in target joints 17. Consumption of rIX-FP - number of rIX-FP infusions (doses) 18. Consumption of rIX-FP - IU/kg per subject per month 19. Consumption of rIX-FP - IU/kg per subject per year 20. Number of bleeding episodes requiring rIX-FP to achieve hemostasis 21. Percentage of bleeding episodes requiring rIX-FP to achieve hemostasis 22. Number of subjects who develop antibodies against rIX-FP 23. Number of subjects who develop antibodies against Chinese hamster ovary host cell protein 24. The number of subjects with treatment emergent adverse events (TEAEs) related to rIX-FP 25. The percentage of subjects with treatment emergent adverse events (TEAEs) related to rIX-FP;Timepoint(s) of evaluation of this end point: 1, 2, 3, 4, 5, 6 and 7. Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK) 8. Before, and at 30 minutes after the end of, rIX-FP infusion at Week 26 9, 10, 11 and 12. Before, and up to 336 hours after the end of, rIX-FP infusion at Week 26 13, 14, 15, 17, 18, 19, 20, 21, 24 and 25. Up to 18 months after rIX-FP infusion 16, 22 and 23. At baseline and up to 18 months after rIX-F | — |
Countries
China
Contacts
Public ContactTrial Registration Coordinator
CSL Behring GmbH
Outcome results
None listed