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

NCT06399289 | Active Not Recruiting Phase 3

• 2 other identifiers: CSL654_30042022-002333-34
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 7

Brief Summary

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.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (7)

• Incremental recovery (IR) (plasma FIX activity)

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

• Maximum plasma concentration (Cmax)

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

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

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

• Area under the concentration-time curve (AUC)

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

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

• Clearance (Cl) of rIX-FP

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

• Annualized spontaneous bleeding rate (AsBR)

  AsBR for treated bleeding episodes, by prophylaxis regimen and overall

  Up to 18 months

• Number of subjects who develop an inhibitor to FIX

  Up to 18 months after rIX-FP infusion

Secondary Outcomes (25)

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

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

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

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

• Mean residence time (MRT)

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

• Apparent volume of distribution during the terminal phase (Vz)

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

• Apparent volume of distribution at steady-state (Vss)

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

Study Arms (1)

rIX-FP

EXPERIMENTAL

Subjects will receive rIX-FP as an intravenous (IV) infusion for a minimum of 50 exposure days (EDs)

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

Interventions

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

Lyophilized powder for solution for intravenous injection

Also known as: rIX-FP, CSL654, Idelvion

rIX-FP

Eligibility Criteria

• Age: Up to 70 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \- 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

You may not qualify if:

• \- 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.

Sponsors & Collaborators

• CSL Behring: lead

Study Sites (7)

Beijing Children's Hospital

Beijing, Beijing Municipality, 100045, China

Location

Union Hospital Affiliated to Fujian Medical University

Fuzhou, Fujian, 350001, China

Location

Nanfang Hospital of Southern Medical University

Guangzhou, Guangdong, 510515, China

Location

Affiliated Hospital of Guizhou Medical University

Guiyang, Guizhou, 550004, China

Location

North China University Of Science And Technology Affiliated Hospital

Tangshan, Hebei, 63000, China

Location

Jinan Central Hospital

Jinan, Shandong, 250013, China

Location

Hospital of Hematology, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

Location

MeSH Terms

Conditions

Hemophilia B

Interventions

Albumins; albutrepenonacog alfa

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Proteins; Amino Acids, Peptides, and Proteins

Study Officials

• Clinical Program Director

  CSL Behring

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 1, 2024
• First Posted: May 3, 2024
• Study Start: July 28, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: January 8, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Time Frame: Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.
• Access Criteria: Proposed research should seek to answer a previously unanswered important medical or scientific question. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.

Locations

CN (7)