Von Willebrand DiseasePhase 3NCT01224808
Extension Study of Biostate in Subjects With Von Willebrand Disease
A Phase 3 study testing Biostate in people with Von Willebrand Disease, run by CSL Behring at 6 sites in 5 countries.
Trial summary
The aim of the Von Willebrand Disease (VWD) therapy is to treat and prevent bleeding episodes due to abnormal platelet adhesion and abnormal blood coagulation as a result of low or abnormal Von Willebrand Factor (VWF) and/or Factor VIII (FVIII) levels. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Von Willebrand Disease
- What is being tested?
- Biostate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 6 sitesin 5 countries
- Study dates
- Oct 2010to Mar 2014
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Have completed Study CSLCT-BIO-08-52 (Assessment of Efficacy and Safety of Biostate in Paediatric Subjects with Von Willebrand Disease) or Study CSLCT-BIO-08-54 (Assessment of Efficacy and Safety of Biostate in Adolescent or Adult Subjects with Von Willebrand Disease).
- The subject and/or his/her legal guardian understand(s) the nature of the study and has/have given written informed consent to participate in the study and is/are willing to comply with the protocol.
Who cannot join
- Early discontinuation of a subject from the main studies CSLCT-BIO-08-52 or CSLCT-BIO-08-54.
- Mental condition rendering the subject (or the subject's legal guardian) unable to understand the nature, scope and possible consequences of the study.
- Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study.
- Are not willing and/or not able to comply with the study requirements.
- Employee at the study site, or spouse/partner or relative of the Investigator or Subinvestigators.
- Female subjects of childbearing potential either not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study, or not sexually abstinent for the entire duration of the study, or not surgically sterile.
- Intention to become pregnant during the course of the study.
- Pregnancy, or nursing mother.
Trial locations and contacts
6 sites in 5 countries.
6 sites in 5 countries
About this study
Official title: An Open-Label, Multi-Centre Extension Study to Assess the Efficacy and Safety of Biostate® in Paediatric, Adolescent, and Adult Subjects With Von Willebrand Disease Who Completed Clinical Studies CSLCT-BIO-08-52 or CSLCTBIO-08-54
The aim of the Von Willebrand Disease (VWD) therapy is to treat and prevent bleeding episodes due to abnormal platelet adhesion and abnormal blood coagulation as a result of low or abnormal Von Willebrand Factor (VWF) and/or Factor VIII (FVIII) levels. The long-term efficacy and safety of a VWF/FVIII concentrate, Biostate, will be investigated in children, adolescents, and adults with VWD in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.
- Conditions
- Von Willebrand Disease
- Being tested
- Biostate
- Keywords
- von Willebrand Disease
- Medical subject headings
- von Willebrand Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BiostateBiological
Single bolus doses, administered intravenously. Frequency and dose will be determined by the Investigator based on the subjects clinical condition, previous VWF concentrate requirements, response to therapy, weight and reason for usage.
| Study group | Role | What they receive |
|---|---|---|
| Experimental | Experimental | Not described Receives: Biological: Biostate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Haemostatic efficacy
Measured: Up to 32 months
Secondary outcomes (8)
Development of FVIII inhibitors
Measured: Up to 32 months
Development of VWF inhibitors
Measured: Up to 32 months
Frequency of Adverse events (AEs) per subject
Measured: 32 months
Severity of AEs per subject
Measured: 32 months
Severity of AEs per infusion
Measured: 32 months
Causality of AEs per subject
Measured: 32 months
Causality of AEs per infusion
Measured: 32 months
Frequency of Adverse events (AEs) per infusion
Measured: 32 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2010 (estimated)
- Study startOct 2010
- Primary completionMar 2014
- Study completionMar 2014
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.
- More trials run by CSL BehringThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Program Director, Clinical R&D, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01224808
- Sponsor's protocol ID
- CSLCT-BIO-09-64
- Other IDs
- 2009-017301-11, 1498, 2009
- Status last verified
- Oct 2017
- First submitted
- 8 Jun 2010
- Last updated
- 3 Oct 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multi-Centre Extension Study to Assess the Efficacy and Safety of Biostate® in Paediatric, Adolescent, and Adult Subjects With Von Willebrand Disease Who Completed Clinical Studies CSLCT-BIO-08-52 or CSLCTBIO-08-54
- ClinicalTrials.gov ID
- NCT01224808
- Acronym / sponsor ID
- CSLCT-BIO-09-64
- Conditions
- Von Willebrand Disease
- Keywords
- von Willebrand Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Oct 2010
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 20 Oct 2010 (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 aim of the Von Willebrand Disease (VWD) therapy is to treat and prevent bleeding episodes due to abnormal platelet adhesion and abnormal blood coagulation as a result of low or abnormal Von Willebrand Factor (VWF) and/or Factor VIII (FVIII) levels. The long-term efficacy and safety of a VWF/FVIII concentrate, Biostate, will be investigated in children, adolescents, and adults with VWD in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental | Experimental | Biological: Biostate |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Have completed Study CSLCT-BIO-08-52 (Assessment of Efficacy and Safety of Biostate in Paediatric Subjects with Von Willebrand Disease) or Study CSLCT-BIO-08-54 (Assessment of Efficacy and Safety of Biostate in Adolescent or Adult Subjects with Von Willebrand Disease).
- The subject and/or his/her legal guardian understand(s) the nature of the study and has/have given written informed consent to participate in the study and is/are willing to comply with the protocol.
Key exclusion criteria (8 listed)
- Early discontinuation of a subject from the main studies CSLCT-BIO-08-52 or CSLCT-BIO-08-54.
- Mental condition rendering the subject (or the subject's legal guardian) unable to understand the nature, scope and possible consequences of the study.
- Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study.
- Are not willing and/or not able to comply with the study requirements.
- Employee at the study site, or spouse/partner or relative of the Investigator or Subinvestigators.
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 |
|---|---|---|
| BiostateSingle bolus doses, administered intravenously. Frequency and dose will be determined by the Investigator based on the subjects clinical condition, previous VWF concentrate requirements, response to therapy, weight and reason for usage. | Biological | Experimental |
Primary outcome
Haemostatic efficacy
Time frame: Up to 32 months
8 secondary outcomes
Development of FVIII inhibitors
Time frame: Up to 32 months
Development of VWF inhibitors
Time frame: Up to 32 months
Frequency of Adverse events (AEs) per subject
Time frame: 32 months
Severity of AEs per subject
Time frame: 32 months
Severity of AEs per infusion
Time frame: 32 months
Causality of AEs per subject
Time frame: 32 months
Causality of AEs per infusion
Time frame: 32 months
Frequency of Adverse events (AEs) per infusion
Time frame: 32 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Oct 2017.
Related trials for Von Willebrand Disease
Active, not recruitingObservational study
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RecruitingObservational study
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HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
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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.
Program Director, Clinical R&D study director
CSL Behring
6 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Biostate in people with Von Willebrand Disease, run by CSL Behring at 6 sites in 5 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 2 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: finished, as of the sponsor's last update on 3 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Poland, Bulgaria, Germany, Russia, Ukraine. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Haemostatic efficacy (time frame: Up to 32 months). 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 Oct 2017.