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vatreptacog alfa

Phase 3

Congenital Bleeding Disorder | Small molecule | Rare Disease |Novo Nordisk A/S|Last Updated: May 15, 2017

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
RandomizedDouble-BlindCONTROLLEDBiomarker
Total Trials2
Total Enrollment104
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01392547Efficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and InhibitorsPHASE3 COMPLETED 72 — — Jul 1, 2011Aug 1, 2012May 15, 201732 United States, Austria +17
NCT00822185Safety, Tolerability and Pharmacokinetics of NN1731 in Healthy VolunteersPHASE1 COMPLETED 32 — — Jan 1, 2009Jul 1, 2009Jan 5, 20151 Japan
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Study Endpoints
Primary Endpoints
Effective Bleeding Control Defined as no Additional Haemostatic Medication (Other Than Trial Product) Given
Within 12 hours of first trial product administration
Safety (Physical Examination, Vital Signs, ECG, Haematology, Biochemistry, Urinalysis, Coagulation Factors, Coagulation-related Parameters, Injection Site Tolerability and Adverse Events (AE))
between dosing and 2-3 weeks after dosing

Any safety issue was reported as AE

Subjects With Anti-Vatreptacog Alfa Antibody
between dosing, 2-3 weeks after dosing, and 11-13 weeks after dosing

Post-dosing samples from subjects were evaluated for the presence of Anti-Vatreptacog alfa antibody

Secondary Endpoints
Effective and Sustained Bleeding Control
Up to 48 hours after first trial product administration
Number of Doses of Trial Product Given for Each Acute Bleed
Up to 6 hours after first trial product administration
Number of Adverse Events
Adverse events were captured from the time of consent to 1 month (+14 days) after last administration of trial product.
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
rFVIIaEXPERIMENTAL -
vatreptocog alfaEXPERIMENTAL -
vatreptacog alfa, 5 mcg/kgEXPERIMENTAL -
vatreptacog alfa, 10 mcg/kgEXPERIMENTAL -
vatreptacog alfa, 20 mcg/kgEXPERIMENTAL -
vatreptacog alfa, 30 mcg/kgEXPERIMENTAL -
Interventions
NameTypeDescription
vatreptacog alfa (activated)DRUG1-3 doses per bleeding episode
eptacog alfa (activated)DRUG1-3 doses per bleeding episode
placeboDRUGSingle dose is injected i.v. over 2 minutes to 2 subjects per dose level: 5 mcg/kg
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Eligibility Criteria
Age Range12 Years — N/A
SexMALE
Healthy VolunteersNo
Study Sites32

Inclusion Criteria: * Male patient with clinical diagnosis of congenital haemophilia A or B and inhibitors to coagulation factors VIII or IX * Minimum of five bleeds requiring haemostatic drug treatment within the previous 12 months at trial entry Exclusion Criteria: * Previous participation in t...

Countries:United StatesAustriaBrazilCroatiaGreeceHungaryItalyJapanMalaysiaPolandPuerto RicoRomaniaRussiaSerbiaSouth AfricaTaiwanThailandTurkey (Türkiye)United Kingdom
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