Clinical trial NCT01361126: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Hemophilia B
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2011-05-26
Enrollment: 17
Sex: MALE
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Number of Subjects With Treatment-related Adverse Events
timeFrame: Approximately 20 weeks
measure: Number of Subjects With Inhibitors Against Factor IX (FIX)
timeFrame: Baseline, Day 10 and Weeks 4, 12 and 20
measure: Number of Subjects Who Developed Antibodies to rIX-FP
timeFrame: Pre-dose, Day 10 and Weeks 4, 12, and 20
Secondary outcomes:
measure: Area Under the Curve to the Last Sample With Quantifiable Drug Concentration (AUC0-t) After a Single Dose of rIX-FP
timeFrame: Pre-dose and up to 14 days after rIX-FP infusion.
measure: Half-life (t1/2) of a Single Dose of rIX-FP
timeFrame: Pre-dose and up to 14 days after infusion
measure: Incremental Recovery of rIX-FP at 30 Minutes Following Infusion of rIX-FP
timeFrame: 30 minutes after infusion
measure: Clearance of a Single Dose of rIX-FP
timeFrame: Pre-dose and up to 14 days after rIX-FP infusion
measure: Breakthrough Bleeding Events
timeFrame: Week 9 to approximately Week 20
Other outcomes:
Locations:
city: Sofia
country: Bulgaria
facility: Study Site
city: Tel Aviv
country: Israel
facility: Study Site
Study officials:
affiliation: CSL Behring
name: Iris Jacobs, MD
role: STUDY_DIRECTOR
References: