Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease
NCT ID: NCT00816660
Last Updated: 2021-05-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1
32 participants
INTERVENTIONAL
2008-12-01
2010-08-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
CROSSOVER
OTHER
SINGLE
Study Groups
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1
Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII)
Single dose, dose escalation, various cohorts
2
Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII)
Single dose, dose escalation, various cohorts
Marketed plasma-derived VWF/FVIII concentrate
Cross-over: recombinant FVIII (rVWF:rFVIII) and marketed plasma-derived VWF/FVIII concentrate
Interventions
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Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII)
Single dose, dose escalation, various cohorts
Marketed plasma-derived VWF/FVIII concentrate
Cross-over: recombinant FVIII (rVWF:rFVIII) and marketed plasma-derived VWF/FVIII concentrate
Eligibility Criteria
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Inclusion Criteria
* 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 least 25 exposure days to VWF/FVIII coagulation factor concentrates
* The subject has a Karnofsky score \>= 70%
* The subject is between 18 to 60 years of age (on the day of signing the informed consent)
* NOT APPLICABLE IN ITALY: Female subjects of child-bearing potential must have a negative pregnancy test and agree to practice contraception using a method of proven reliability from the day of screening until the study completion visit
* APPLICABLE ONLY IN ITALY: Female subjects of child-bearing potential must have a negative pregnancy test and agree to practice non-hormonal-based contraception using a method of proven reliability (IUD acceptable) from the day of screening until 96 hours after the last investigational drug infusion
* NOT APPLICABLE IN ITALY: The subject must agree not to be on any therapy (hormone-based contraception acceptable) interfering with coagulation factor pharmacokinetics until 96 hours after the last investigational drug infusion
* APPLICABLE ONLY IN ITALY: The subject must agree not to be on any therapy interfering with coagulation factor pharmacokinetics until 96 hours after the last investigational drug infusion
Exclusion Criteria
* 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 known hypersensitivity to mouse or hamster proteins
* The subject has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, food allergies or animal allergies
* The subject has a medical history of a thromboembolic event
* The subject is HIV positive with an absolute CD4 count \< 200/mm3
* The subject has been diagnosed with cardiovascular disease (New York Heart Association (NYHA) classes 1-4)
* The subject has been diagnosed with insulin-dependent diabetes mellitus
* The subject has an acute illness (e.g. influenza, flu-like syndrome, allergic rhinitis/conjunctivitis)
* The subject has been diagnosed with liver disease, as evidenced by, but not limited to, any of the following: serum ALT three times the upper limit of normal, hypoalbuminemia, portal vein hypertension (e.g. presence of otherwise unexplained splenomegaly, history of esophageal varices)
* The subject has been diagnosed with renal disease, with a serum creatinine level \>= 2 mg/dL
* In the judgment 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 has been treated with an immunomodulatory drug, excluding topical treatment (e.g. ointments, nasal sprays) within 30 days before enrollment
* The subject has been treated with drugs known to induce thrombotic thrombocytopenic purpura (TTP) (e.g. Adenosine diphosphate (ADP) receptor inhibitors (Clopidogrel, Ticlopidine)) within 60 days before enrollment
* The subject is receiving or anticipates receiving another investigational and/or interventional drug within 30 days before enrollment
* The subject is a lactating female
* The subject has a history of drug or alcohol abuse within the last 5 years
* The subject has a progressive fatal disease and/or life expectancy of less than 3 months
* The subject is identified by the investigator as being unable or unwilling to cooperate with study procedures
* The subject suffers from a mental condition rendering him/her unable to understand the nature, scope and possible consequences of the study and/or evidence of an uncooperative attitude
* Subject is in prison or compulsory detention by regulatory and/or juridical order
18 Years
60 Years
ALL
No
Sponsors
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Baxalta now part of Shire
INDUSTRY
Responsible Party
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Principal Investigators
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Study Director
Role: STUDY_DIRECTOR
Takeda
Locations
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Emory University School of Medicine, Dept. of Pediatrics
Atlanta, Georgia, United States
Rush University Medical Center
Chicago, Illinois, United States
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States
University of Kentucky Hemophilia Treatment Center
Lexington, Kentucky, United States
Brown Cancer Center
Louisville, Kentucky, United States
Brigham & Women´s Hospital, Hematology Division
Boston, Massachusetts, United States
Rochester General Hospital
Rochester, New York, United States
Hemophilia Center of Western PA
Pittsburgh, Pennsylvania, United States
University of Texas
Houston, Texas, United States
Comprehensive Center for Bleeding Disorders
Milwaukee, Wisconsin, United States
General Hospital Vienna (Allgemeines Krankenhaus der Stadt Wien), University Department for Internal Medicine I
Vienna, , Austria
Q.E.II Health Sciences Centre
Halifax, Nova Scotia, Canada
Vivantes Klinikum im Friedrichshain
Berlin, , Germany
Hannover Medical School - Clinic for Haematology, Haemostaseology, Oncology and Stem Cell Transplantation
Hanover, , Germany
Institut für Thrombophilie und Hämostaseologie
Münster, , Germany
Azienda Ospedaliero-universitaria "Careggi"
Florence, , Italy
Giannia Gaslini Children´s Hospital
Genova, , Italy
Ospedale Maggiore di Milano, Centro Emofilia e Trombosi "Angelo Bianchi Bonomi"
Milan, , Italy
Ospedale San Giovanni Bosco, Centro Emofilia Divisione di Ematologia
Naples, , Italy
University of Padua Medical School
Padua, , Italy
Ospedale di Vicenza - U.L.S.S.N.6
Vicenza, , Italy
West Midlands Region Adult Haemophilia Centre, Queen Elizabeth Hospital
Birmingham, , United Kingdom
Imperial College School of Medicine, Hammersmith Hospital
London, , United Kingdom
Central Manchester Healthcare NHS Trust, Manchester Haemophilia Comprehensive Care Centre
Manchester, , United Kingdom
Royal Cornwall Hospital
Truro, , United Kingdom
Countries
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References
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Mannucci PM, Kempton C, Millar C, Romond E, Shapiro A, Birschmann I, Ragni MV, Gill JC, Yee TT, Klamroth R, Wong WY, Chapman M, Engl W, Turecek PL, Suiter TM, Ewenstein BM; rVWF Ad Hoc Study Group. Pharmacokinetics and safety of a novel recombinant human von Willebrand factor manufactured with a plasma-free method: a prospective clinical trial. Blood. 2013 Aug 1;122(5):648-57. doi: 10.1182/blood-2013-01-479527. Epub 2013 Jun 18.
Other Identifiers
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070701
Identifier Type: -
Identifier Source: org_study_id
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