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Marketed plasma-derived VWF/FVIII

Phase 1

Von Willebrand Disease | Monoclonal antibody | Hematology |Takeda Pharmaceutical Company Limited|Last Updated: May 3, 2021

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
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00816660Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand DiseasePHASE1 COMPLETED 32Dec 1, 2008Aug 31, 2010May 3, 202125 United States, Austria +4
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Study Endpoints
Primary Endpoints
To demonstrate the immediate tolerability and safety after single-dose injections of rVWF:rFVIII at various doses
Up to 30 days after the last investigational product infusion
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelCROSSOVER
PurposeOTHER
Treatment Arms
ArmTypeDescription
1EXPERIMENTAL -
2ACTIVE_COMPARATOR -
Interventions
NameTypeDescription
Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII)BIOLOGICALSingle dose, dose escalation, various cohorts
Marketed plasma-derived VWF/FVIII concentrateBIOLOGICALCross-over: recombinant FVIII (rVWF:rFVIII) and marketed plasma-derived VWF/FVIII concentrate
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Eligibility Criteria
Age Range18 Years — 60 Years
SexALL
Healthy VolunteersNo
Study Sites25

Inclusion Criteria: * Subject has voluntarily given written informed consent (before conduct of any study-related procedures) * The subject has hereditary type 3 VWD (\<= 3 IU/dL VWF:Ag)or severe type 1 or type 2A VWD (VWF:RCo \<= 10% and FVIII:C \<20%) * The subject has a medical history of at lea...

Countries:United StatesAustriaCanadaGermanyItalyUnited Kingdom
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