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

Phase 1

Conditions: von Willebrands Disease
MedDRA version: 9.1Level: LLTClassification code 10047715Term: Von Willebrand's disease

Registration Number: EUCTR2006-000663-28-PL

Lead Sponsor: Bio Products Laboratory

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: All

Target Recruitment: 26

Inclusion Criteria

1. Have given written informed consent.
2. Be aged 12 years or older.
3. Have severe VWD (VWF:RCo <20%) of known type. Severity will be confirmed by a current VWF:RCo result of <20%.
4. Be known or expected to require a concentrate for management of VWD.
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 DDAVP, or for whom DDAVP is contraindicated.
7. Have a prothombin time (PT) of not more than 3 seconds above the upper limit of the reference range.
8. At the Baseline Visit (Visit 1), patients must have had at least 5 days since their last infusion of replacement factor concentrate or DDAVP (see section 9.3.10 regarding the use of other concomitant medication).
9. Female patients of child-bearing potential must have a negatice result on a human chorionic gonadotropin-based pregnancy test. If a female patient is or becomes sexually active, she must practice contraception by using a method of proven reliability for the duration of the study. Female patients must not be lactating.
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) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

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.
8. In the option of the investigator, the pateint is unlikely to comply with the study protocol.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Pharmacokinetics of Optivate;Secondary Objective: Efficacy and Safety of Optivate in long-term use over at least 12 months.;Primary end point(s): AUC (0-t), AUC(0-72h), MRT(0-t) and MRT (0-72h) for VWF:RCo at the Baseline Visit (Visit 1) by VWD type and overall

Secondary Outcome Measures

Name	Time	Method

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