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A Study With OPTIVATE® in People With Von Willebrand Disease

Primary Purpose

Von Willebrand Disease

Status

Terminated

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Optivate

About this trial

This is an interventional treatment trial for Von Willebrand Disease focused on measuring Von Willebrand Disease, Factor VIII, Von Willebrand Factor, Pharmacokinetics

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Have given written informed consent.
Be aged 12 years or older.
Have severe VWD (VWF:RCo <20%) of known type. Severity will be confirmed by a current VWF:RCo result of <20%.
Be known or expected to require a concentrate for management of VWD.
Must have had at least one bleed in the last 12 months which required treatment with a FVIII and VWF concentrate.
Have a known lack of, or poor response to, DDAVP.
Have a prothrombin time (PT) of not more than 3 seconds above the upper limit of the reference range.
At the Baseline Visit (Visit 1), patients must have had at least 5 days since their last infusion of replacement factor concentrate or DDAVP.
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:

Have a history of inhibitor development to VWF or FVIII or a positive result at screening.
Actively bleeding (Note: the patient can enter the study once the bleed is controlled).
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.
Known or suspected hypersensitivity to investigational medicinal product (IMP) or its excipients.
Have a recent history of alcohol or drug abuse.
Administration of a new chemical entity within the 4 months preceding enrolment.
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 the opinion of the investigator, the patient is unlikely to comply with the study protocol.

Sites / Locations

Rambam Health Care Campus, 8 Haaliya St., Bat-Galim
Haddasah Ein-Karem Medical Center, P.O.Box 12000
Beilinson Hospital, Rabin Medical Center, 39 Jabontinsky Street
University Department of Haematology

Outcomes

Primary Outcome Measures

Pharmacokinetic parameters for VWF RCo at the Baseline Visit by VWD type and overall.

Secondary Outcome Measures

Full Information

NCT ID

NCT00387192

First Posted

October 11, 2006

Last Updated

March 2, 2010

Sponsor

Bio Products Laboratory

1. Study Identification

Unique Protocol Identification Number

NCT00387192

Brief Title

A Study With OPTIVATE® in People With Von Willebrand Disease

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2010

Overall Recruitment Status

Terminated

Why Stopped

Due to slow recruitment and a significant delay in reaching the recruitment target.

Study Start Date

November 2006 (undefined)

Primary Completion Date

undefined (undefined)

Study Completion Date

September 2008 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

Bio Products Laboratory

4. Oversight

Data Monitoring Committee

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Von Willebrand Disease

Keywords

Von Willebrand Disease, Factor VIII, Von Willebrand Factor, Pharmacokinetics

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

26 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

Optivate

Intervention Description

Plasma-derived Factor VIII

Primary Outcome Measure Information:

Title

Pharmacokinetic parameters for VWF RCo at the Baseline Visit by VWD type and overall.

Time Frame

Baseline vist

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Have given written informed consent. Be aged 12 years or older. Have severe VWD (VWF:RCo <20%) of known type. Severity will be confirmed by a current VWF:RCo result of <20%. Be known or expected to require a concentrate for management of VWD. Must have had at least one bleed in the last 12 months which required treatment with a FVIII and VWF concentrate. Have a known lack of, or poor response to, DDAVP. Have a prothrombin time (PT) of not more than 3 seconds above the upper limit of the reference range. At the Baseline Visit (Visit 1), patients must have had at least 5 days since their last infusion of replacement factor concentrate or DDAVP. 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: Have a history of inhibitor development to VWF or FVIII or a positive result at screening. Actively bleeding (Note: the patient can enter the study once the bleed is controlled). 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. Known or suspected hypersensitivity to investigational medicinal product (IMP) or its excipients. Have a recent history of alcohol or drug abuse. Administration of a new chemical entity within the 4 months preceding enrolment. 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 the opinion of the investigator, the patient is unlikely to comply with the study protocol.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Charles Hay, MD

Organizational Affiliation

Manchester Haemophilia Comprehensive Care Centre

Official's Role

Principal Investigator

Facility Information:

Facility Name

Rambam Health Care Campus, 8 Haaliya St., Bat-Galim

City

Haifa

ZIP/Postal Code

31096

Country

Israel

Facility Name

Haddasah Ein-Karem Medical Center, P.O.Box 12000

City

Jerusalem

ZIP/Postal Code

91120

Country

Israel

Facility Name

Beilinson Hospital, Rabin Medical Center, 39 Jabontinsky Street

City

Petah Tikva

ZIP/Postal Code

49100

Country

Israel

Facility Name

University Department of Haematology

City

Manchester

ZIP/Postal Code

M13 9WL

Country

United Kingdom

12. IPD Sharing Statement

Links:

URL

http://www.bpl.co.uk

Description

Sponsor's homepage

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A Study With OPTIVATE® in People With Von Willebrand Disease

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