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

A Phase II/III Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects with Hemophilia B

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-002415-28-DE

Enrollment

Registered

2011-06-24

Start date

2012-01-19

Completion date

Unknown

Last updated

2015-11-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia B MedDRA version: 16.1 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850

Interventions

Product Name: Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) Product Code: CSL654 Pharmaceutical Form: Powder for concentrate for solution for infusion Current Sponsor

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: • Male subjects, 12 to 65 years old. • Severe hemophilia B (FIX activity of = 2%). • Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs). • No history of FIX inhibitor formation, no detectable inhibitors at Screening and no family history of inhibitors against FIX. • Written informed consent for study participation. • On-demand subjects only, who have experienced a minimum average of 2 non-trauma induced bleeding episodes requiring treatment with a FIX product during the previous 6 or 3 months. Are the trial subjects under 18? yes Number of subjects for this age range: 3 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 55 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2

Exclusion criteria

Exclusion criteria: • Known hypersensitivity to any FIX product or hamster protein. • Known congenital or acquired coagulation disorder other than congenital FIX deficiency. • HIV positive subjects with a CD4 count < 200/mm3. • Low platelet count, kidney or liver dysfunction. • Recent life-threatening bleeding episode.

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objectives of the study are to evaluate the efficacy of rIX-FP in preventing bleeding episodes (prophylaxis) and safety of rIX-FP with respect to the development of inhibitors to FIX in patients with severe hemophilia B. ;Secondary Objective: The secondary objectives of the study are: •To evaluate the safety of rIX-FP, based on AEs and the development of antibodies to rIX-FP. •To evaluate the clinical response to rIX-FP for the prevention and treatment of bleeding episodes in patients with severe hemophilia B. •To evaluate the efficacy of rIX-FP in surgical prophylaxis. •To evaluate the pharmacokinetics (PK) of a single dose of rIX-FP. ;Primary end point(s): Change in frequency of spontaneous bleeding events between ondemand and prophylaxis treatments (annualized);Timepoint(s) of evaluation of this end point: approximately 14 months	—

Measure

Time frame

Main Objective: The primary objectives of the study are to evaluate the efficacy of rIX-FP in preventing bleeding episodes (prophylaxis) and safety of rIX-FP with respect to the development of inhibitors to FIX in patients with severe hemophilia B. ;Secondary Objective: The secondary objectives of the study are: •To evaluate the safety of rIX-FP, based on AEs and the development of antibodies to rIX-FP. •To evaluate the clinical response to rIX-FP for the prevention and treatment of bleeding episodes in patients with severe hemophilia B. •To evaluate the efficacy of rIX-FP in surgical prophylaxis. •To evaluate the pharmacokinetics (PK) of a single dose of rIX-FP. ;Primary end point(s): Change in frequency of spontaneous bleeding events between ondemand and prophylaxis treatments (annualized);Timepoint(s) of evaluation of this end point: approximately 14 months

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Secondary

Measure	Time frame
Secondary end point(s): - The frequency of related Adverse Events (AEs) to rIX-FP over the course of the study. - The number of subjects with FIX inhibitors. - The number of subjects with antibodies against rIX-FP. - Proportion of bleeding episodes requiring one or = two infusions of rIX-FP to achieve hemostasis. - Investigator’s overall clinical assessment of hemostatic efficacy for treatment of bleeding episodes, based on a four point ordinal scales (excellent, good, moderate, poor/ none). - rIX-FP consumed per month while maintaining assigned prophylactic treatment interval during routine prophylaxis. - Incremental recovery (IU/mL/IU/kg) at 30 minutes following infusion of rIX-FP. - Half-life (t1/2) of a single dose of rIX-FP. - AUC to the last sample with quantifiable drug concentration (AUC0-t) of a single dose of rIX-FP. - Clearance of a single dose of rIX-FP. - Annualized spontaneous bleeding events during the 7 day prophylactic regimen period compared to the annualized spontaneous bleeding events during each of the prophylactic regimens longer than the 7 day prophylactic regimen period. - Investigator’s (or surgeon’s) overall clinical assessment of hemostatic efficacy for surgical prophylaxis, based on a four point ordinal scale (excellent, good, moderate, poor/ none);Timepoint(s) of evaluation of this end point: All timepoints are approximately 14 months except for the PK endpoints, which are 240 hours or as indicated in the endpoint description, and the Investigator's overall clinical assessment of hemostatic efficacy for surgical prophylaxis, which is approximately 14 days.	—

Measure

Time frame

Secondary end point(s): - The frequency of related Adverse Events (AEs) to rIX-FP over the course of the study. - The number of subjects with FIX inhibitors. - The number of subjects with antibodies against rIX-FP. - Proportion of bleeding episodes requiring one or = two infusions of rIX-FP to achieve hemostasis. - Investigator’s overall clinical assessment of hemostatic efficacy for treatment of bleeding episodes, based on a four point ordinal scales (excellent, good, moderate, poor/ none). - rIX-FP consumed per month while maintaining assigned prophylactic treatment interval during routine prophylaxis. - Incremental recovery (IU/mL/IU/kg) at 30 minutes following infusion of rIX-FP. - Half-life (t1/2) of a single dose of rIX-FP. - AUC to the last sample with quantifiable drug concentration (AUC0-t) of a single dose of rIX-FP. - Clearance of a single dose of rIX-FP. - Annualized spontaneous bleeding events during the 7 day prophylactic regimen period compared to the annualized spontaneous bleeding events during each of the prophylactic regimens longer than the 7 day prophylactic regimen period. - Investigator’s (or surgeon’s) overall clinical assessment of hemostatic efficacy for surgical prophylaxis, based on a four point ordinal scale (excellent, good, moderate, poor/ none);Timepoint(s) of evaluation of this end point: All timepoints are approximately 14 months except for the PK endpoints, which are 240 hours or as indicated in the endpoint description, and the Investigator's overall clinical assessment of hemostatic efficacy for surgical prophylaxis, which is approximately 14 days.

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Countries

Austria, Bulgaria, France, Germany, Israel, Italy, Japan, Russian Federation, Spain, United States

Contacts

Public ContactGlobal Project Leader

CSL Behring

grace.cole@cslbehring.com+16108784751

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026