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
18 Years
65 Years
Both
Von Willebrand Disease
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 Hospital | Philadelphia, Pennsylvania 19107 |
| Newark Beth Israel Medical Center | Newark, New Jersey 07112 |
| University of California Davis Cancer Center | Sacramento, California 95817 |
| University of Illinois College of Medicine at Peoria | Peoria, Illinois 61603 |
| University of North Carolina at Chapel Hill | Chapel Hill, North Carolina 27599 |
| University Of Miami Miller School Of Medicine | Miami, Florida 33010 |
| Indiana Hemophilia and Thrombosis Center | Indianapolis, Indiana 46260 |
| Blood Center of Wisconsin | Milwaukee, Wisconsin |
| The Mary M Gooley Hemophilia Center and Rochester General Hospital | Rochester, New York 14621 |
| Hemophilia Center of Western Pennsylvania | Pittsburgh, Pennsylvania 15213 |
