Phase 3 N=25 Treatment

Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B

Hemophilia B

Bottom Line

View on ClinicalTrials.gov: NCT01335061 ↗

Enrolled (actual)

Serious AEs

10.0%

Results posted

May 2015

Primary outcome: Primary: Annualized Number of Bleeding Episodes. — 32.9; 3.6 Number of bleeds per year — p=<0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Nonacog alfa (Biological)
Age: Pediatric, Adult, Older Adult · 12+ yrs
Sex: Male
Sponsor: Pfizer
Primary completion: Apr 2014

Outcome Measures

Outcome	Result	p-value
PRIMARY Annualized Number of Bleeding Episodes.	32.9; 3.6	<0.0001 sig
SECONDARY Response to On-Demand Treatment for All Bleeding Episodes.	271; 39; 177; 80; 55; 33	—
SECONDARY Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode.	416; 69; 9; 3; 10	—
SECONDARY Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX.	1.0	—
SECONDARY Average Infusion Dose.	52; 99	—
SECONDARY Total Factor Consumption.	707; 4985	—
SECONDARY Incidence of Less Than Expected Therapeutic Effect (LETE)	0; 0; 0	—

Summary

The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.

Eligibility Criteria

Inclusion Criteria

Documented history of moderately-severe to severe hemophilia B (FIX activity ULN (upper limit of normal) of the reporting laboratory.
Subjects with a known hypersensitivity to any FIX product or hamster protein.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01335061). 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.