A Study With OPTIVATE® in People With Von Willebrand Disease

Von Willebrand Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00387192
• Other study ID #: 8VWF01
• Status: Terminated
• Phase: Phase 3
• First received: October 11, 2006
• Last updated: March 2, 2010
• Start date: November 2006
• Est. completion date: September 2008

Study information

• Verified date: March 2010
• Source: Bio Products Laboratory
• Contact: n/a
• Is FDA regulated: No
• Health authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial Summary

The main objective of the study is to assess the pharmacokinetics of OPTIVATE® after a single dose of 80 IU/kg VWF:RCo. The secondary objectives of the study are to assess efficacy and safety of OPTIVATE® in long-term use over at least 12 months.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 26
• Est. completion date: September 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years and older

Eligibility

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 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.

7. Have a prothrombin 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.

9. Female patients of child-bearing potential must have a negative 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.

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.

8. In the opinion of the investigator, the patient is unlikely to comply with the study protocol.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Von Willebrand Disease
• Von Willebrand Diseases

Intervention

Drug:
• Optivate
Plasma-derived Factor VIII

Locations

Country	Name	City	State
Israel	Rambam Health Care Campus, 8 Haaliya St., Bat-Galim	Haifa
Israel	Haddasah Ein-Karem Medical Center, P.O.Box 12000	Jerusalem
Israel	Beilinson Hospital, Rabin Medical Center, 39 Jabontinsky Street	Petah Tikva
United Kingdom	University Department of Haematology	Manchester

Sponsors (1)

Lead Sponsor	Collaborator
Bio Products Laboratory

Countries where clinical trial is conducted

Israel,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Pharmacokinetic parameters for VWF RCo at the Baseline Visit by VWD type and overall.		Baseline vist

External Links

•   Sponsor's homepage