A safety and Efficacy Study of a recombinant Factor IX in patients with severe Hemophilia B

Active, not recruiting

• Conditions: Prophylaxis and treatment of bleeding episodes in subjects with congenital Factor IX (FIX) deficiency (Hemophilia B).; MedDRA version: 14.0Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2011-002415-28-ES
• Lead Sponsor: CSL Behring GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-10-28
• Last Update Posted: 7 October 2014

Recruitment & Eligibility

• Status: Not Recruiting
• Sex: Male
• Target Recruitment: 60

Inclusion Criteria

- 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

Exclusion Criteria

- 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/?L 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