Von Willebrand DiseasePhase 3NCT00387192
A Study With OPTIVATE® in People With Von Willebrand Disease
A Phase 3 study testing Optivate in people with Von Willebrand Disease, run by Bio Products Laboratory at 4 sites in 2 countries.
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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Von Willebrand Disease
- What is being tested?
- Optivate
- How many people
- 26 participants
- Where
- 4 sitesin 2 countries
- Study dates
- Nov 2006to Sep 2008
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
Israel3 sites
Rambam Health Care Campus, 8 Haaliya St., Bat-Galim
Haifa, 31096
Haddasah Ein-Karem Medical Center, P.O.Box 12000
Jerusalem, 91120
Beilinson Hospital, Rabin Medical Center, 39 Jabontinsky Street
Petah Tikva, 49100
United Kingdom1 site
University Department of Haematology
Manchester, M13 9WL
About this study
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
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.
- Conditions
- Von Willebrand Disease
- Being tested
- Optivate
- Keywords
- Von Willebrand Disease, Factor VIII, Von Willebrand Factor, Pharmacokinetics
- Medical subject headings
- von Willebrand Diseases, Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OptivateDrug
Plasma-derived Factor VIII
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetic parameters for VWF RCo at the Baseline Visit by VWD type and overall.
Measured: Baseline vist
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2006 (estimated)
- Study startNov 2006
- Study completionSep 2008
Why it stopped: Due to slow recruitment and a significant delay in reaching the recruitment target.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bio Products Laboratory
- Sponsor type
- University, hospital or other
- Investigators
- Charles Hay, MD, Principal investigator, Manchester Haemophilia Comprehensive Care Centre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00387192
- Sponsor's protocol ID
- 8VWF01
- Status last verified
- Mar 2010
- First submitted
- 11 Oct 2006
- Last updated
- 3 Mar 2010
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00387192
- Acronym / sponsor ID
- 8VWF01
- Conditions
- Von Willebrand Disease
- Keywords
- Von Willebrand Disease, Factor VIII, Von Willebrand Factor, Pharmacokinetics
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to slow recruitment and a significant delay in reaching the recruitment target.
- Enrolment
- 26 participants (estimated)
- Lead sponsor
- Bio Products LaboratoryUniversity, hospital or other
- Study start
- Nov 2006
- Study completion
- Sep 2008 (actual)
- First posted
- 12 Oct 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OptivatePlasma-derived Factor VIII | Drug | – |
Primary outcome
Pharmacokinetic parameters for VWF RCo at the Baseline Visit by VWD type and overall.
Time frame: Baseline vist
Results and safety
The study was stopped early (reason given: Due to slow recruitment and a significant delay in reaching the recruitment target.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2010.
- Sponsor's homepageLink provided by the sponsor.
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Von Willebrand Diseases
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Charles Hay, MD principal investigator
Manchester Haemophilia Comprehensive Care Centre
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Optivate in people with Von Willebrand Disease, run by Bio Products Laboratory at 4 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 3 Mar 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Israel, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Due to slow recruitment and a significant delay in reaching the recruitment target.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pharmacokinetic parameters for VWF RCo at the Baseline Visit by VWD type and overall. (time frame: Baseline vist). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Mar 2010.