A safety and Efficacy Study of a recombinant Factor IX in patients with severe Hemophilia B
- Conditions
- Prophylaxis and treatment of bleeding episodes in subjects with congenital Factor IX (FIX) deficiency (Hemophilia B).MedDRA version: 14.1Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Body processes [G] - Genetic Phenomena [G05]
- Registration Number
- EUCTR2011-002415-28-IT
- Lead Sponsor
- CSL BEHRING GMBH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 60
Male subjects, 12 to 65 years of age. - Documented severe hemophilia B (FIX activity of = 2%), or confirmed at Screening by the central laboratory. - Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs), confirmed by their treating physician. - No confirmed prior history of FIX inhibitor formation (defined as two consecutive positive tests –requiring a confirmatory test on a second separately drawn blood sample shortly after the previous positive test), no confirmed detectable inhibitors (defined as < 0.6 Bethesda Units [BU]) at Screening by the central laboratory, and no family history of inhibitor formation against FIX. - Written informed consent for study participation obtained before undergoing any study specific procedures. For on-demand subjects ONLY: - Subjects who have experienced a minimum average of 2 non-trauma induced bleeding episodes per month in the past 3 to 6 months, which required FIX replacement therapy and are documented in their medical records. - Subjects who are willing to switch to a prophylaxis regimen.
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
- 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 IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment. - Platelet count < 100,000/µL at Screening. - HIV positive subjects with a CD4 count < 200/mm3. An HIV-positive subject may participate in the study and receive antiviral therapy at the discretion of the Investigator. - Serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT) concentration > 5 x ULN at Screening. - Serum creatinine concentration > 2 x ULN at Screening. - Evidence of thrombosis, including deep vein thrombosis, stroke, myocardial infarction or arterial embolus within 4 months prior to dosing on Day 1. - Experienced a life-threatening bleeding episode, including bleeding in the central nervous system, gastrointestinal tract, neck/throat or severe trauma-induced bleeding episode, or had major surgical intervention within 4 months prior to dosing on Day 1. - Use of any Investigational Medicine Product (IMP) other than rIX-FP within 4 weeks prior to the first rIX-FP administration on Day 1. - Concurrent non-hemophiliac inflammatory joint disease or other medical condition that, in the Investigator's judgment, could confound study results. - Suspected inability (e.g., language problem or mental condition) or unwillingness to comply with study procedures or history of noncompliance. - Subjects who have active synovitis. - Subject routinely receives factor IX infusion prior to activity (ie, sports) as a preventative measure more than 2 times per month.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method