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

Completed · Phase 1

Conditions studied: Von Willebrand Disease

In brief

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.

Key facts

Study ID

NCT00816660

Run by

Baxalta now part of Shire

People needed

32

Starts

2008-12-01

Expected to finish

2010-08-31

Last updated by the study team

2021-05-03

Who can join

Age: 18 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 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

You may not qualify if…

• The subject has been diagnosed with a hereditary or acquired coagulation disorder other than VWD (including qualitative and quantitative platelet disorders and/or an international normalized ratio (INR) > 1.4)
• 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

Where it is running

• 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

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.