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Recombinant von Willebrand factor: recombinant FVIII

Phase 1

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

Target and mechanism

Molecular targetF9
Target classExogenous Protein
ModalityMonoclonal antibody

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLEDDMC
Total Trials1
Total Enrollment32

FDA Designations

No designations recorded

Clinical trial landscape

Recombinant von Willebrand factor: recombinant FVIII · 1 trial · 1 indication

Phase 1 1
NCT00816660Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand DiseaseVon Willebrand Disease
COMPLETED32 Analytics
PHASE1COMPLETED
Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease
Von Willebrand DiseaseUnlock trial analytics

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 to 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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Competitive Landscape -Von Willebrand Disease 8 trials

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Frequently asked questions about Recombinant von Willebrand factor: recombinant FVIII

What is recombinant von Willebrand factor: recombinant FVIII?

Recombinant von Willebrand factor: recombinant FVIII is an investigational biologic complex being studied for Von Willebrand Disease, a bleeding disorder. It combines recombinant von Willebrand factor with recombinant factor VIII. Takeda Pharmaceutical Company Limited is developing it, and it has been tested in a Phase 1 pharmacokinetic, safety and tolerability study.

What is recombinant von Willebrand factor: recombinant FVIII used for in Von Willebrand Disease?

It is being studied for use in Von Willebrand Disease, specifically in patients with Type 3 Von Willebrand Disease in the completed Phase 1 trial. The study evaluated pharmacokinetics, safety and tolerability of the recombinant von Willebrand factor and recombinant factor VIII complex in adults aged 18 years and older.

What does recombinant von Willebrand factor: recombinant FVIII target?

The complex targets F9, also known as factor IX, and is classified as an exogenous protein therapy. It supplies recombinant von Willebrand factor alongside recombinant factor VIII. The completed Phase 1 study examined how this complex behaved pharmacokinetically in patients with Type 3 Von Willebrand Disease.

Who is developing recombinant von Willebrand factor: recombinant FVIII?

Takeda Pharmaceutical Company Limited, which trades under the ticker TAK, is the developer of recombinant von Willebrand factor: recombinant FVIII. The company sponsored the completed Phase 1 trial of the complex in Type 3 Von Willebrand Disease.

What phase is recombinant von Willebrand factor: recombinant FVIII in?

Recombinant von Willebrand factor: recombinant FVIII is in Phase 1 development and is investigational. One Phase 1 trial has been completed, and no trials are currently active. The completed study enrolled 32 participants and was randomized, active controlled, and conducted without blinding.

What clinical trials is recombinant von Willebrand factor: recombinant FVIII in?

The complex has been studied in one completed Phase 1 trial, NCT00816660, titled a pharmacokinetic, safety and tolerability study of recombinant von Willebrand factor and recombinant factor VIII complex in Type 3 Von Willebrand Disease. It enrolled 32 adults across the United States, Austria, Canada, Germany, Italy, and the United Kingdom.

Is recombinant von Willebrand factor: recombinant FVIII the same as rVWF:rFVIII?

Yes, rVWF:rFVIII is another name for recombinant von Willebrand factor: recombinant FVIII. The abbreviation stands for the recombinant von Willebrand factor and recombinant factor VIII complex. Both names refer to the same investigational product developed by Takeda for Von Willebrand Disease.