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A Phase 3 Clinical Study to Determine the Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor : Recombinant Factor VIII (rVWF:rFVIII) and rVWF in the Treatment of Bleeding Episodes in Subjects Diagnosed With Von Willebrand Disease


Phase 3
18 Years
65 Years
Open (Enrolling)
Both
Von Willebrand Disease

Thank you

Trial Information

A Phase 3 Clinical Study to Determine the Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor : Recombinant Factor VIII (rVWF:rFVIII) and rVWF in the Treatment of Bleeding Episodes in Subjects Diagnosed With Von Willebrand Disease


Selected

Inclusion Criteria:



- The subject has been diagnosed with type 3 von Willebrand disease (VWD:Ag <= 3 IU/dl)
or severe non-type 3 VWD (VWF:RCo < 20 IU/dL) or type 2N VWD (FVIII:C <10% and
historically documented genetics)

- The subject, who participates for the treatment of bleeding episodes, has had a
minimum of 6 documented bleeds (medical history) requiring VWF coagulation factor
replacement therapy during the previous 3 years prior to enrollment

- The subject is at least 18 and not older than 65 years of age at enrollment

Selected Exclusion Criteria:

- The subject has been diagnosed with pseudo VWD or another hereditary or acquired
coagulation disorder other than VWD (e.g., qualitative and quantitative platelet
disorders or elevated PT/international normalized ratio [INR] > 1.4)

- The subject has a documented history of a VWF:RCo half-life of < 6 hours

- The subject has a history or presence of a VWF inhibitor at screening

- The subject has a history or presence of a factor VIII (FVIII) inhibitor with a titer
>= 0.4 BU (by Nijmegen assay) or >= 0.6 BU (by Bethesda assay)

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of subjects with treatment success for treated bleeding episodes

Outcome Description:

Treatment success will be defined as a mean efficacy rating score of < 2.5 for a subject´s bleeding episodes treated with the investigational product while in a treatment period. Scores used to assess the extent of control of the bleeding episodes: Excellent = 1, Good = 2, Moderate = 3, None = 4.

Outcome Time Frame:

12 months

Safety Issue:

No

Principal Investigator

Benny Sorensen, MD

Investigator Role:

Study Director

Investigator Affiliation:

Baxter Healthcare Corporation

Authority:

Australia: Department of Health and Ageing Therapeutic Goods Administration

Study ID:

071001

NCT ID:

NCT01410227

Start Date:

September 2011

Completion Date:

March 2014

Related Keywords:

  • Von Willebrand Disease
  • Hemorrhage
  • Von Willebrand Diseases

Name

Location

Pennsylvania HospitalPhiladelphia, Pennsylvania  19107
Newark Beth Israel Medical CenterNewark, New Jersey  07112
University of California Davis Cancer CenterSacramento, California  95817
University of Illinois College of Medicine at PeoriaPeoria, Illinois  61603
University of North Carolina at Chapel HillChapel Hill, North Carolina  27599
University Of Miami Miller School Of MedicineMiami, Florida  33010
Indiana Hemophilia and Thrombosis CenterIndianapolis, Indiana  46260
Blood Center of WisconsinMilwaukee, Wisconsin  
The Mary M Gooley Hemophilia Center and Rochester General HospitalRochester, New York  14621
Hemophilia Center of Western PennsylvaniaPittsburgh, Pennsylvania  15213