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

Conditions: Hemophilia B
MedDRA version: 16.1Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 100000004850
Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Registration Number: EUCTR2011-002415-28-DE

Lead Sponsor: CSL Behring GmbH

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 60

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

• 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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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:<br>•To evaluate the safety of rIX-FP, based on AEs and the development of antibodies to rIX-FP.<br>•To evaluate the clinical response to rIX-FP for the prevention and treatment of bleeding episodes in patients with severe hemophilia B.<br>•To evaluate the efficacy of rIX-FP in surgical prophylaxis.<br>•To evaluate the pharmacokinetics (PK) of a single dose of rIX-FP.<br><br><br>;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

Secondary Outcome Measures