A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B

Phase 3

Completed

• Conditions: Hemophilia B

• Registration Number: NCT02053792
• Lead Sponsor: CSL Behring

Brief Summary

This study will examine the long-term safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children and adults with severe hemophilia B. The study will include subjects who have not previously been treated with Factor IX products, subjects who previously completed a CSL-sponsored rIX-FP lead-in study and subjects requiring major non-emergency surgery who have not previously completed a CSL-sponsored rIX-FP lead-in study.

A surgical prophylaxis substudy will examine the efficacy of rIX-FP in subjects with hemophilia B who are undergoing non-emergency major or minor surgery. An additional substudy will examine the safety and PK of subcutaneous (SC) administration of rIX-FP.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-01-29
• Study First Submitted QC: 2014-02-02
• Study First Posted: 2014-02-04
• Study Start: 2014-02-06
• Primary Completion: 2021-06-02
• Study Completion: 2021-06-02
• Results First Submitted: 2022-05-17
• Status Verified: 2022-07
• Results First Submitted QC: 2022-07-12
• Last Update Submitted: 2022-07-12
• Results First Posted: 2022-07-14
• Last Update Posted: 2022-07-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 97

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Mean Incremental Recovery of a 50 IU/kg Dose of CSL654 in Previously Untreated Patients (PUPs)	Approximately 30 minutes after infusion of CSL654	Incremental Recovery: The increase in plasma concentration per IU/kg of factor administered.
Total Number of Participants Who Developed Inhibitors Against Factor IX (FIX)	For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs.

Secondary Outcome Measures

Name	Time	Method
Total Annualized Bleeding Rate (ABR) by Prophylaxis Regimen in Previously Treated Patients (PTPs)	For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Spontaneous ABR by Prophylaxis Regimen in PTPs	For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Number of Participants With Investigator's Overall Clinical Assessment of Hemostatic Efficacy for the Treatment of Major Bleeding Events With CSL654 in PUPs	Up to 3 years or the time it takes to achieve 50 EDs	The investigator will rate the efficacy of the rIX-FP treatment based on a hemostatic efficacy four point rating scale of excellent, good, moderate, or poor/no response.
Total ABR for On-demand Regimen vs. 14-Day Regimen in PTPs	For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Spontaneous ABR for On-demand Regimen vs. 14-Day Regimen in PTPs	For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Total ABR for Subjects >=12 Years: 7-Day Regimen vs. 14-Day Regimen in PTPs	For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Average Amount of CSL654 (rIX-FP) Consumed Per Month Per Subject During Routine Prophylaxis Treatment.	For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs.
Spontaneous ABR for Subjects >=12 Years: 7-Day Regimen vs. (10 or 14)-Day Regimen in PTPs	For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Percentage of Participants With at Least One Treatment Emergent Adverse Event (TEAE) and the Percentage of Participants With at Least One CSL654-related TEAE	For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs.
Spontaneous ABR for Subjects >=12 Years: 7-Day Regimen vs. 14-Day Regimen in PTPs	For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Total ABR for Subjects >=12 Years: 7-Day Regimen vs. (10 or 14)-Day Regimen in PTPs	For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).

Trial Locations

Locations (42)

University of Colorado; 🇺🇸 Aurora, Colorado, United States

Indiana Hemophilia & Thrombosis Center Inc.; 🇺🇸 Indianapolis, Indiana, United States

Hospital of the University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

University of Utah; 🇺🇸 Salt Lake City, Utah, United States

Royal Children's Hospital; 🇦🇺 Parkville, Victoria, Australia

The Children's Hospital Westmead; 🇦🇺 Westmead, Victoria, Australia

Department of Pediatrics, Medical University of Vienna; 🇦🇹 Vienna, Austria

Medical University of Vienna, Vienna General Hospital; 🇦🇹 Vienna, Austria

SHAT "Joan Pavel" ODD [Hemorrhagic Diathesis & Anemia]; 🇧🇬 Sofia, Bulgaria

McMaster University; 🇨🇦 Hamilton, Ontario, Canada

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