Recent Updates
Recently added Catalysts

Recombinant Coagulation Factor IX

Phase 3

Hemophilia B | Small molecule | Hematology |Pfizer, Inc.|Last Updated: Oct 4, 2011

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedCONTROLLED
Total Trials1
Total Enrollment50
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00364182Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia BPHASE3 COMPLETED 50May 1, 2007Oct 1, 2010Oct 4, 201119 United States, Canada +7
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Annualized Number of Bleeding Episodes
Baseline up to Week 56

Annualized bleed rate (ABR) or number of bleeds per year derived for each participant for each treatment regimen by using the following formula: ABR = number of bleeds / (days on treatment regimen / 365.25)

Secondary Endpoints
Amount of Sleep Measured by Sleep Diary After Hemarthrosis
24 and 48 hours post-bleed
Quality of Sleep Measured by Sleep Diary After Hemarthrosis
24 and 48 hours post-bleed
Acute Pain After Hemarthrosis
24 and 48 hours post-bleed
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelCROSSOVER
PurposePREVENTION
Treatment Arms
ArmTypeDescription
AEXPERIMENTAL -
BEXPERIMENTAL -
Interventions
NameTypeDescription
Recombinant Coagulation Factor IX (BeneFIX)DRUG100 IU/kg once weekly then crossover to 50 IU/kg twice weekly
Unlock Study Design Details
Eligibility Criteria
Age Range6 Years — 65 Years
SexMALE
Healthy VolunteersNo
Study Sites19

Inclusion Criteria: * Documented history of moderately severe or severe hemophilia B (FIX:C less than or equal to 2%) * Male subjects, aged 6 years to 65 years. * Subjects with a minimum of 12 bleeding episodes, 6 of which must be joint bleeds, in the 12 months before screening. Exclusion Criteria...

Countries:United StatesCanadaCroatiaHungaryItalyRomaniaRussiaSerbiaSpain
Unlock Eligibility Criteria