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

Status: Recruiting
Phase:
Diagnosis: Other Trials
NCT ID: NCT00816660 (View complete trial on ClinicalTrials.gov)
DFCI Protocol ID: 09-239

 

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.

 

Conducting Institutions:
Brigham and Women's Hospital, Dana-Farber Cancer Institute

Overall PI:
Judith Lin, MD, Brigham and Women's Hospital

Site-responsible Investigators:

Contacts:

Eligibility Criteria

Inclusion Criteria: - 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 Exclusion Criteria: - 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
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