Phase 3
N=63
A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
Hemophilia B
Bottom Line
View on ClinicalTrials.gov: NCT01496274 ↗Enrolled (actual)
63
Serious AEs
4.8%
Results posted
May 2016
Primary outcome: Primary: Change in Frequency of Spontaneous Bleeding Events Between On-demand and Prophylaxis Treatments (Annualized) — 15.43; 0.00 bleeds/year/subject — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- rIX-FP (Biological)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- Male
- Sponsor
- CSL Behring
- Primary completion
- Jul 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Frequency of Spontaneous Bleeding Events Between On-demand and Prophylaxis Treatments (Annualized) |
15.43; 0.00 | <0.0001 sig |
| PRIMARY Number of Subjects Developing Inhibitors Against Factor IX (FIX) |
0; 0 | — |
| SECONDARY The Frequency of Related Adverse Events |
10.0; 4.3; 7.9; 87.5; 78.3; 84.1 | — |
| SECONDARY Number of Subjects Developing Antibodies Against rIX-FP |
— | — |
| SECONDARY Proportion of Bleeding Episodes Requiring One or ≤ Two Injections of rIX-FP to Achieve Hemostasis |
92.1; 94.5; 91.9; 100.0; 98.6; 94.6 | — |
| SECONDARY Investigator's Overall Clinical Assessment of Hemostatic Efficacy for Treatment of Bleeding Episodes, Based on a Four Point Ordinal Scales (Excellent, Good, Moderate, Poor/No Response) |
71.3; 87.5; 20.8; 7.4; 3.0; 2.3 | — |
| SECONDARY rIX-FP Consumed Per Month While Maintaining Assigned Prophylactic Treatment Interval During Routine Prophylaxis. |
191.69; 202.68; 201.50; 157.44 | — |
| SECONDARY Incremental Recovery of rIX-FP |
1.29; 1.24 | — |
| SECONDARY Half-life (t1/2) of a Single Dose of rIX-FP |
104.77; 96.88 | — |
| SECONDARY Area Under the Curve (AUC) |
6534.15; 5963.30 | — |
| SECONDARY Clearance of a Single Dose of rIX-FP |
50.19; 59.00 | — |
| SECONDARY Investigator's (or Surgeon's) Overall Clinical Assessment of Hemostatic Efficacy for Surgical Prophylaxis, Based on a Four Point Ordinal Scale (Excellent, Good, Moderate, Poor/No Response) |
6; 0; 0; 0 | — |
| SECONDARY Annualized Spontaneous Bleeding Events Compared Between 7 Day Prophylactic and Extended Regimens |
0.00; 0.00; 0.00 | — |
Summary
This study will examine the safety, pharmacokinetics and efficacy of rIX-FP for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B.
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
- On-demand subjects only, who have experienced a minimum average of 2 non-trauma induced bleeding episodes requiring treatment with a FIX product during the previous 6 or 3 months
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, kidney or liver dysfunction
- Recent life-threatening bleeding episode
Data sourced from ClinicalTrials.gov (NCT01496274). 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.