A Phase 3 clinical study to determine the pharmacokinetics, safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding episodes in subjects diagnosed with von Willebrand disease
- Conditions
- 1006447710005330Coagulation disorderHereditary deficiency of Von Willebrand Factor in blood
- Registration Number
- NL-OMON38008
- Lead Sponsor
- Baxter
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 4
The 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
• The 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 previous 12 months prior to enrollment.
• The subject has a Karnofsky score >=60.
• The subject is at least 18 and not older than 65 years of age at enrollment.
• The 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).
• The subject has a documented history of a VWF:RCo half-life of <6 hours.
• The subject has a history or presence of a VWF inhibitor at screening.
• The 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).
• The subject has a known hypersensitivity to any of the components of the study drugs, such as to
mouse or hamster proteins.
• The subject has a medical history of immunological disorders, excluding seasonal allergic
rhinitis/conjunctivitis, mild asthma, 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 an acute illness (eg, influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, non-seasonal asthma) at screening.
• The 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).
• 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 (eg, uncontrolled hypertension) that may pose additional risks for the subject.
• The subject has been treated with an immunomodulatory drug, excluding topical treatment (eg, ointments, nasal sprays), within 30 days prior to signing the informed consent.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Number of subjects with a treatment success for treated bleeding episodes</p><br>
- Secondary Outcome Measures
Name Time Method