Brief Title
A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B
Official Title
A Phase I/II Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B
Brief Summary
This study will examine the safety and efficacy of a Recombinant Coagulation Factor IX
Albumin Fusion Protein (rIX-FP) for the control and prevention of bleeding episodes in
subjects who have previously received factor replacement therapy for hemophilia B. The study
consists of a screening period, a pharmacokinetic (PK) period, followed by approximately a 5
month treatment period. Subjects will receive weekly routine prophylactic therapy and
on-demand treatment for bleeding episodes. In addition, subjects who are not on routine
factor replacement therapy prior to the study will receive only on-demand treatment for
bleeding episodes.
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Number of Subjects With Treatment-related Adverse Events
Secondary Outcome
Area Under the Curve to the Last Sample With Quantifiable Drug Concentration (AUC0-t) After a Single Dose of rIX-FP
Condition
Hemophilia B
Intervention
Recombinant Coagulation Factor IX Albumin Fusion Protein
Study Arms / Comparison Groups
On-demand
Description: The routine prophylactic therapy interval is targeted at every 7 days.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
17
Start Date
July 2011
Completion Date
July 2012
Primary Completion Date
June 2012
Eligibility 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 obtained before undergoing any study
specific procedures
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, abnormal kidney function, or liver disease
- On-demand subjects experiencing less than 12 or 6 non-trauma induced bleeding episodes
requiring treatment with a FIX product during the previous 6 or 3 months, respectively
- Planned major surgical intervention during the study period
Gender
Male
Ages
12 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Iris Jacobs, MD, ,
Location Countries
Bulgaria
Location Countries
Bulgaria
Administrative Informations
NCT ID
NCT01361126
Organization ID
CSL654_2004
Secondary IDs
2010-023793-39
Responsible Party
Sponsor
Study Sponsor
CSL Behring
Study Sponsor
Iris Jacobs, MD, Study Director, CSL Behring
Verification Date
April 2016