Phase 2
N=17
A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B
Hemophilia B
Bottom Line
View on ClinicalTrials.gov: NCT01361126 ↗Enrolled (actual)
17
Serious AEs
0.0%
Results posted
May 2016
Primary outcome: Primary: Number of Subjects With Treatment-related Adverse Events — 0 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Recombinant Coagulation Factor IX Albumin Fusion Protein (Biological)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- Male
- Sponsor
- CSL Behring
- Primary completion
- Jun 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Subjects With Treatment-related Adverse Events |
— | — |
| PRIMARY Number of Subjects With Inhibitors Against Factor IX (FIX) |
— | — |
| PRIMARY Number of Subjects Who Developed Antibodies to rIX-FP |
— | — |
| SECONDARY Area Under the Curve to the Last Sample With Quantifiable Drug Concentration (AUC0-t) After a Single Dose of rIX-FP |
2915; 2960 | — |
| SECONDARY Half-life (t1/2) of a Single Dose of rIX-FP |
77.7; 138 | — |
| SECONDARY Incremental Recovery of rIX-FP at 30 Minutes Following Infusion of rIX-FP |
1.47; 1.35 | — |
| SECONDARY Clearance of a Single Dose of rIX-FP |
0.749; 0.699 | — |
| SECONDARY Breakthrough Bleeding Events |
0.2 | — |
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
Data sourced from ClinicalTrials.gov (NCT01361126). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.