Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease - Full Text View

Sponsored by:	Baxter Healthcare Corporation
Information provided by:	Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:	NCT00816660

Purpose

The objectives of this study are to evaluate the immediate tolerability and safety of rVWF:rFVIII in subjects with Type 3 Von Willebrand Disease after administration of various dosages of VWF:RCo.

Condition	Intervention	Phase
Von Willebrand Disease	Biological: Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII) Biological: Marketed plasma-derived VWF/FVIII concentrate	Phase I

Study Type:	Interventional
Study Design:	Randomized, Single Blind (Subject), Active Control, Crossover Assignment, Safety Study
Official Title:	Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex (rVWF:rFVIII): A Phase 1 Study Evaluating the Pharmacokinetics (PK), Safety, and Tolerability in Type 3 Von Willebrand Disease (VWD)

Resource links provided by NLM:

Genetics Home Reference related topics: hemophilia von Willebrand disease

Drug Information available for: Octocog alfa Moroctocog Alfa Factor VIII

U.S. FDA Resources

Further study details as provided by Baxter Healthcare Corporation:

Primary Outcome Measures:

To demonstrate the immediate tolerability and safety after various doses of rVWF:rFVIII. [ Time Frame: approximately up to 9 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	31
Study Start Date:	December 2008
Estimated Primary Completion Date:	March 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental	Biological: Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII) Single dose, dose escalation, various cohorts
2: Active Comparator	Biological: Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII) Single dose, dose escalation, various cohorts Biological: Marketed plasma-derived VWF/FVIII concentrate Cross-over: recombinant FVIII (rVWF:rFVIII) and marketed plasma-derived VWF/FVIII concentrate

Eligibility

Ages Eligible for Study:	18 Years to 60 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria (selected):

The subject has hereditary type 3 Von Willebrand Disease (VWD)
The subject is between 18 to 60 years of age.
Female subjects of child-bearing potential must have a negative pregnancy test and agree to practice contraception using a method of proven reliability for the duration of their participation in the study (hormone-based contraception acceptable).

Exclusion Criteria (selected):

The subject has been diagnosed with a hereditary or acquired coagulation disorder other than VWD.
The subject has been diagnosed with an ADAMTS13 deficiency with less than 10% ADAMTS13 activity.
The subject has a history or presence of VWF inhibitor.
The subject has a history or presence of FVIII inhibitor with a titer >= 0.4 BU (by Nijmegen assay) or >= 0.6 BU (by Bethesda assay).
The subject has a medical history of a thromboembolic event.
In the judgement of the investigator, the subject has another clinically significant concomitant disease (e.g. uncontrolled hypertension, diabetes type II) that may pose additional risks for the subject.
The subject is a lactating female.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00816660

Locations

United States, Indiana
Indiana Hemophilia and Thrombosis Center	Recruiting
Indianapolis, Indiana, United States, 46260
Contact: Brandy Trawinski, RN 317-871-0000 btrawinski@IHTC.org
Principal Investigator: Amy Shapiro, MD
United States, Pennsylvania
Hemophilia Center of Western PA	Not yet recruiting
Pittsburgh, Pennsylvania, United States, 15213-4306
Contact: Kristen Jaworski, RN, BSN, CCRC 412-209-7284 kjaworski@itxm.org
Principal Investigator: Margaret Ragni, MD
United States, Texas
Georgetown University Medical Center	Not yet recruiting
Houston, Texas, United States, 77030
Contact: Nidra Rodriguez, MD 713-500-8360 Nidra.I.Rodriguez@uth.tmc.edu
Contact: Madeline Cantini 713-500-8360 Kathryn.A.Bradley@uth.tmc.edu
United States, Wisconsin
Comprehensive Center for Bleeding Disorders	Not yet recruiting
Milwaukee, Wisconsin, United States, 53225-3548
Contact: Joan Gill, MD 414-257-2424 Joan.Gill@bcw.edu
Contact: Megan Gavin 713-500-8376 Megan.Gavin@bcw.edu
Principal Investigator: Joan Gill, MD
Austria
General Hospital Vienna (Allgemeines Krankenhaus der Stadt Wien), University Department for Internal Medicine I	Recruiting
Vienna, Austria, 1090
Contact: Ingrid Pabinger, MD +43-1-40400-4448 ingrid.pabinger@meduniwien.ac.at
Principal Investigator: Ingrid Pabinger, MD
Italy
Ospedale San Giovanni Bosco, Centro Emofilia Divisione di Ematologia	Not yet recruiting
Naples, Italy, 80144
Contact: Angela Rocino, MD +39-81-254-5299
Principal Investigator: Angela Rocino, MD
Ospedale Maggiore di Milano, Centro Emofilia e Trombosi "Angelo Bianchi Bonomi"	Not yet recruiting
Milan, Italy, 20122
Contact: Pier M. Mannucci, MD +39-02-5503-5305
Principal Investigator: Pier M. Mannucci, MD
United Kingdom
Central Manchester Healthcare NHS Trust, Manchester Haemophilia Comprehensive Care Centre	Not yet recruiting
Manchester, United Kingdom, M13 9WL
Contact: Charles R Hay, MD +44-161-276-3360 charles.hay@manchester.ac.uk
Principal Investigator: Charles R Hay, MD
Queen Mary´s School of Medicine and Dentistry, Centre of Haemostasis and Thrombosis	Not yet recruiting
London, United Kingdom, E1 2AT
Contact: John Pasi, MD +44-207-377-7455 k.j.pasi@qmul.ac.uk
Principal Investigator: John Pasi, MD
West Midlands Region Adult Haemophilia Centre, Queen Elizabeth Hospital	Not yet recruiting
Birmingham, United Kingdom, B15 2TT
Contact: Will Lester, MD +44-121-627-2353 will.lester@uhb.nhs.uk
Principal Investigator: Will Lester, MD
Royal Free Hospital, Haemophilia Centre and Thrombosis Unit	Not yet recruiting
London, United Kingdom, NW3 2QG
Contact: Thynn Thynn Yee, MD +44-20-7794-0500 ext 35317 Sonia.Rotoloni@royalfree.nhs.uk
Principal Investigator: Thynn Thynn Yee, MD
Imperial College School of Medicine, Hammersmith Hospital	Not yet recruiting
London, United Kingdom, W12 0NN
Contact: Michael Laffan, MD +44-208-383-2178 m.laffan@imperial.ac.uk
Principal Investigator: Michael Laffan, MD

Sponsors and Collaborators

Baxter Healthcare Corporation

Investigators

Study Director:

Tobias Suiter, MD

Baxter Healthcare Corporation

More Information

No publications provided

Responsible Party:	Baxter Healthcare Corporation ( Tobias Suiter, MD; Medical Director )
Study ID Numbers:	070701
Study First Received:	January 2, 2009
Last Updated:	May 6, 2009
ClinicalTrials.gov Identifier:	NCT00816660 History of Changes
Health Authority:	United States: Food and Drug Administration; Austria: Federal Ministry for Health Family and Youth; Italy: Ministry of Health; United Kingdom: Medicines and Healthcare Products Regulatory Agency

Study placed in the following topic categories:

Von Willebrand Disease
Thrombocytopathy
Von Willebrand Disease, Recessive Form
Hemorrhagic Disorders
Genetic Diseases, Inborn

Hematologic Diseases
Blood Platelet Disorders
Blood Coagulation Disorders
Hemostatic Disorders
Factor VIII

Additional relevant MeSH terms:

Von Willebrand Disease
Coagulants
Hematologic Diseases
Coagulation Protein Disorders
Blood Coagulation Disorders
Blood Platelet Disorders
Hematologic Agents

Factor VIII
Pharmacologic Actions
Blood Coagulation Disorders, Inherited
Hemorrhagic Disorders
Genetic Diseases, Inborn
Therapeutic Uses

ClinicalTrials.gov processed this record on September 02, 2009