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moroctocog alfa

Phase 3

Hemophilia A | Small molecule | Hematology |Pfizer, Inc.|Last Updated: Jan 11, 2019

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedCONTROLLEDDMC
Total Trials2
Total Enrollment82
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00543439Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIIIPHASE3 COMPLETED 66Dec 1, 2007Apr 1, 2018Jan 11, 201928 United States, Argentina +11
NCT01579903Relative Bioavailability Of Two Formulations Of Moroctocog Alfa (AF-CC)PHASE1 COMPLETED 16Aug 1, 2012Jan 1, 2013Feb 8, 20133 Bulgaria, Hungary +1
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Study Endpoints
Primary Endpoints
Mean Annualized Bleed Rate (ABR) by Treatment: On Demand Cohort
Day 1 up to Month 6 (OD Cohort, OD Therapy, Period 1); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2)

ABR for each participant was calculated as the number of bleeds requiring administration of moroctocog alfa (AF-CC) divided by the total therapy duration (in days), then multiplied by 365.25 (days in a year).

Single dose pharmacokinetics of moroctocog alfa (AF-CC) using noncompartmental analysis (primary PK parameters include: Cmax, AUClast, and AUCinf)
Periods 1 and 2, Day 1 through 4
Secondary Endpoints
Mean Annualized Bleed Rate (ABR) by Treatment: Routine Prophylaxis Cohort
Day 1 up to Month 24 (RP Cohort, Period 1 and Period 2)
Mean of Moroctocog Alfa (AF-CC) Infusions Administered To Treat Bleeding Episode: All Participants
Day 1 up to Month 24
Number of Treated Bleeds Classified on Basis of Response to First Infusion of Moroctocog Alfa (AF-CC) as On-Demand Treatment: OD Therapy (OD and RP Cohort)
Day 1 up to Month 24
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
1EXPERIMENTALOn-Demand therapy for 6 months, followed by Routine Prophylaxis treatment for 1 year.
2EXPERIMENTALRoutine Prophylaxis Crossover
Sequence 1EXPERIMENTALSubjects randomized to receive Treatment A during Period 1, then Treatment B during Period 2.
Sequence 2EXPERIMENTALSubjects randomized to receive Treatment B during Period 1, then Treatment B during Period 2.
Interventions
NameTypeDescription
Moroctocog alfa (AF-CC)BIOLOGICALOn-demand therapy for 6 months, followed by routine prophylaxis 25 IU/kg, administered every other day for 1 year.
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Eligibility Criteria
Age Range6 Months — 15 Years
SexMALE
Healthy VolunteersNo
Study Sites28

Inclusion Criteria: * Male subjects, aged less than 6 years, with moderately severe to severe hemophilia A. * A negative FVIII inhibitor titer at screening, and a medical history negative for a past FVIII inhibitor. * At least 20 exposure days to any FVIII replacement product. * Adequate hepatic an...

Countries:United StatesArgentinaAustriaColombiaCroatiaJordanMexicoNew ZealandOmanPeruPolandRomaniaTurkey (Türkiye)BulgariaHungaryUnited Kingdom
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