Clinical study to explore the influence of the human body on the study drug, on the safety and the efficacy of recombinant VWF (in combination with recombinant FVIII and recombinant VWF alone) in the treatment of bleeding episodes in patients with von Willebrand Disease.
- Conditions
- Von Willebrand DiseaseMedDRA version: 14.1Level: PTClassification code 10047715Term: Von Willebrand's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2010-024108-84-AT
- Lead Sponsor
- Baxter Innovations GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 49
• Subject has been diagnosed with:
Type 1 (VWF:RCo < 20 IU/dL) or,
Type 2A (VWF:RCo< 20 IU/dL), Type 2B (as diagnosed by genotype),
Type 2N FVIII:C<10% and historically documented genetics), Type 2M or,
Type 3 (VWF:Ag = 3 IU/dL) or,
Severe VWD with a history of requiring substitution therapy with von Willebrand factor
concentrate to control bleeding
• Subject, who participates for the treatment for bleeding episodes, has
had a minimum of 1 documented bleeds (medical history) requiring VWF coagulation factor replacement therapy during
the previous12 months prior to enrollment.
• Subject has a Karnofsky score =60.
• Subject is at least 18 and not older than 65 years of age at enrollment.
• If female of childbearing potential, subject presents with a negative
serum pregnancy test.
• Subject agrees to employ adequate birth control measures for the
duration of the study.
• Subject is willing and able to comply with the requirements of the protocol.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 49
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
• Subject has been diagnosed with pseudo VWD or another hereditary or
acquired coagulation disorder other than VWD (eg qualitative and
quantitative platelet disorders or elevated PT/ international normalized
ratio [INR] >1.4).
• Subject has a documented history of a VWF:RCo half-life of <6 hours.
• Subject has a history or presence of a VWF inhibitor at screening.
• 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).
• Subject has a known hypersensitivity to any of the components of the
study drugs, such as to mouse or hamster proteins.
• Subject has a medical history of immunological disorders, excluding
seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies or
animal allergies.
• Subject has a medical history of a thromboembolic event.
• Subject is HIV positive with an absolute CD4 count <200/mm3.
• Subject has been diagnosed with cardiovascular disease (New York
Heart Association [NYHA] classes 1-4).
• Subject has an acute illness (eg, influenza, flu-like syndrome, allergic
rhinitis/conjunctivitis, non-seasonal asthma) at screening.
• Subject has been diagnosed with significant liver disease as evidenced
by any of the following: serum alanine aminotransferase (ALT) 5 times
the upper limit of normal; hypoalbuminemia; portal vein hypertension
(eg, presence of otherwise unexplained splenomegaly, history of
esophageal varices).
• Subject has been diagnosed with renal disease, with a serum
creatinine level =2 mg/dL.
• In the judgment of the investigator, subject has another clinically
significant concomitant disease (eg, uncontrolled hypertension) that
may pose additional risks for the subject.
• Subject has been treated with an immunomodulatory drug, excluding
topical treatment (eg, ointments, nasal sprays), within 30 days prior to
signing the informed consent.
• Subject is pregnant or lactating at the time of enrollment.
• Subject has participated in another clinical study involving an IP or
investigational device within 30 days prior to enrollment or is scheduled
to participate in another clinical study involving an investigational
product or investigational device during the course of this study.
• Subject has a history of drug or alcohol abuse within the 2 years prior
to enrollment.
• Subject has a progressive fatal disease and/or life expectancy of less
than 3 months.
• Subject is identified by the investigator as being unable or unwilling to
cooperate with study procedures.
• 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method