An open multi-centre study in patients with von Willebrand Disease to investigate the pharmacokinetics, efficacy and safety of Optivate, a high purity, dual inactivated Factor VIII and Von Willebrand Factor concentrate - A PK study on Optivate in patients with von Willebrands Disease
- Conditions
- von Willebrands Disease
- Registration Number
- EUCTR2006-000663-28-GB
- Lead Sponsor
- Bio Products Laboratory
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 26
1.Given written informed consent.2.Be aged 12 years or older.3.Have severe VWD (VWF:RCo <20%) of known type (confirmed by one previous and one current VWF:RCo result of <20%) .4.Be known or expected to require a concentrate for management.5.Must have had at least one spontaneous bleed in the last 12 months which required treatment with a FVIII and VWF concentrate.6.Have a known lack of, or poor response to, Desmopressin (DDAVP), or for whom DDAVP is contraindicated.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1.Have a history of inhibitor development to VWF or FVIII or a positive result at screening 2.Actively bleeding (Note: the patient can enter the study once the bleed is controlled).3.Presence of major systemic illnesses: renal disease, liver disease, or neurological or psychiatric disease which would compromise the outcome of the study in the opinion of the investigator.4.Known or suspected hypersensitivity to investigational medicinal product (IMP) or its excipients.5.Have a recent history of alcohol or drug abuse.6.Administration of a new chemical entity within the 4 months preceding enrolment.7.Participation in any other clinical study in which investigational or marketed drugs were employed in the 30 days preceding enrolment into this study, with the exception of the BPL clinical study protocol 8VWF03, in such cases patients should have completed their End-of study visit either before or on the day of screening for this study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Pharmacokinetcs of Optivate;Secondary Objective: Efficacy and Safety of Optivate;Primary end point(s): AUC (0-t), AUC9)-72h), mean residence time (MRT0(0-t) and MRT (0-72h) for VWF:RCo at the baseline visit by VWD type and overall
- Secondary Outcome Measures
Name Time Method