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Completed

NCT Number: NCT06399289

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy

This study will investigate the pharmacokinetics (PK), efficacy, and safety of rIX-FP for the routine prophylaxis of bleeding episodes in male Chinese previously treated patients (PTPs) with hemophilia B (FIX activity of ≤ 2%). In addition to the scheduled rIX-FP prophylaxis regimen, subjects may also receive rIX-FP episodic (on-demand) treatment for breakthrough bleeding episodes and rIX-FP for the prophylaxis and treatment of bleeding in emergency surgical procedures.

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Key information

Conditions

Hemophilia B Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

Up to 70 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Beijing Children's Hospital, Beijing, Beijing Municipality, China

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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 < 6 years)
  • Subjects have no confirmed prior history of FIX inhibitor formation

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.

Treatment and study plan

Recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP)

Biological

Lyophilized powder for solution for intravenous injection

Other names: rIX-FP, CSL654, Idelvion

Primary outcomes

  1. Incremental recovery (IR) (plasma FIX activity)

    Time frame: Before, and at 30 minutes after the end of, rIX-FP infusion on Day 1

  2. Maximum plasma concentration (Cmax)

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1

  3. Terminal elimination half-life (t1/2) of rIX-FP

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1

  4. Area under the concentration-time curve (AUC)

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1

    AUC from time zero to the last measurable concentration (plasma FIX activity) (AUC0-last), and AUC from time zero extrapolated to infinity (AUC0-inf).

  5. Clearance (Cl) of rIX-FP

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1

  6. Annualized spontaneous bleeding rate (AsBR)

    Time frame: Up to 18 months

    AsBR for treated bleeding episodes, by prophylaxis regimen and overall

  7. Number of subjects who develop an inhibitor to FIX

    Time frame: Up to 18 months after rIX-FP infusion

Secondary outcomes

  1. Percentage of area under the concentration-time curve extrapolated (%AUCExt)

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK)

  2. Area under the first moment versus time curve extrapolated to infinity (AUMC0-∞)

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK)

  3. Mean residence time (MRT)

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK)

  4. Apparent volume of distribution during the terminal phase (Vz)

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK)

  5. Apparent volume of distribution at steady-state (Vss)

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK)

  6. Time to reach Cmax (Tmax)

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK)

  7. Elimination rate constant (λz)

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion on Day 1 and at Week 26 (Repeat PK)

  8. Incremental recovery (IR) (plasma FIX activity) - Repeat PK

    Time frame: Before, and at 30 minutes after the end of, rIX-FP infusion at Week 26

  9. Maximum plasma concentration (Cmax) - Repeat PK

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion at Week 26

  10. Terminal elimination half-life (t1/2) of rIX-FP - Repeat PK

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion at Week 26

  11. Area under the concentration-time curve (AUC) - Repeat PK

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion at Week 26

    AUC from time zero to the last measurable concentration (plasma FIX activity) (AUC0-last), and AUC from time zero extrapolated to infinity (AUC0-inf).

  12. Clearance (Cl) of rIX-FP - Repeat PK

    Time frame: Before, and up to 336 hours after the end of, rIX-FP infusion at Week 26

  13. Annualized bleeding rate (ABR)

    Time frame: Up to 18 months after rIX-FP infusion

    ABR for treated, untreated, and both treated and untreated bleeding episodes (spontaneous bleeding, traumatic bleeding, unknown bleeding, and total bleeding episodes), by prophylaxis regimen and overall

  14. Annualized joint bleeding rate (AjBR)

    Time frame: Up to 18 months after rIX-FP infusion

    AjBR for treated, untreated, and both treated and untreated bleeding episodes, by prophylaxis regimen and overall

  15. Clinical evaluation of hemostatic efficacy for major bleeding episodes

    Time frame: Up to 18 months after rIX-FP infusion

    The investigator will rate the efficacy of the rIX-FP treatment for major bleeding episodes based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or no efficacy", by prophylaxis regimen and overall

  16. Change in target joints

    Time frame: At baseline and up to 18 months after rIX-FP infusion

  17. Consumption of rIX-FP - number of rIX-FP infusions (doses)

    Time frame: Up to 18 months after rIX-FP infusion

    Consumption of rIX-FP expressed as number of rIX-FP infusions (doses), for the prophylaxis regimens, episodic (on-demand) treatment for bleeding episodes (if any), and total (overall) treatment

  18. Consumption of rIX-FP - IU/kg per subject per month

    Time frame: Up to 18 months after rIX-FP infusion

    Consumption of rIX-FP expressed as total amount (IU/kg) per subject per month, for the prophylaxis regimens, episodic (on-demand) treatment for bleeding episodes (if any), and total (overall) treatment

  19. Consumption of rIX-FP - IU/kg per subject per year

    Time frame: Up to 18 months after rIX-FP infusion

    Consumption of rIX-FP expressed as total amount (IU/kg) per subject per year, for the prophylaxis regimens, episodic (on-demand) treatment for bleeding episodes (if any), and total (overall) treatment.

  20. Number of bleeding episodes requiring rIX-FP to achieve hemostasis

    Time frame: Up to 18 months after rIX-FP infusion

    Number of bleeding episodes requiring 1, ≤ 2, or > 2 infusions (doses) of rIX-FP to achieve hemostasis

  21. Percentage of bleeding episodes requiring rIX-FP to achieve hemostasis

    Time frame: Up to 18 months after rIX-FP infusion

    Percentage of bleeding episodes requiring 1, ≤ 2, or > 2 infusions (doses) of rIX-FP to achieve hemostasis

  22. Number of subjects who develop antibodies against rIX-FP

    Time frame: Before, and up to 18 months after, rIX-FP infusion

  23. Number of subjects who develop antibodies against Chinese hamster ovary host cell protein

    Time frame: Before, and up to 18 months after, rIX-FP infusion

  24. The number of subjects with treatment emergent adverse events (TEAEs) related to rIX-FP

    Time frame: Up to 18 months after rIX-FP infusion

  25. The percentage of subjects with treatment emergent adverse events (TEAEs) related to rIX-FP

    Time frame: Up to 18 months after rIX-FP infusion

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

A Phase 3, Open-label, Multicenter, Pharmacokinetics, Efficacy, and Safety Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Previously Treated Chinese Subjects With Hemophilia B

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
May 3, 2024
Registry last updated
Jul 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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