Von Willebrand DiseasePhase 2/3NCT00941616
Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease
A Phase 2/3 study testing Biostate® in people with Von Willebrand Disease, run by CSL Behring at 5 sites in 4 countries.
Trial summary
The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD). Pharmacokinetic Component: PK parameters will be determined from a subgroup of subjects. 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?
- Biostate®
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 5 sitesin 4 countries
- Study dates
- Jun 2009to Feb 2012
- Phase
- Phase 2/3
- 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
- Diagnosed with VWD
- Desmopressin acetate (DDAVP) treatment is ineffective or contraindicated or not available
- Evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B) within 10 years prior to their first dose of Biostate®
- Written informed consent given
Who cannot join
- Actively bleeding immediately prior to initial PK period
- Have received DDAVP or a VWF product in the 5 days prior to their first dose of study product
- Have Type 2B, 2N or 2M VWD
- Requiring a VWF product for a planned surgical procedure at enrolment
- Have received aspirin or other non-steroidal anti-inflammatory drugs within 7 days prior to their first dose of study product
- Known history of, or are suspected to have, VWF or FVIII inhibitors
Show all 12Show fewer
- Suffering an acute or chronic medical condition, other than VWD, which may affect the conduct of the study
- Known or suspected hypersensitivity or previous evidence of severe side effects to Biostate®, VWF/FVIII concentrates, or human albumin
- Impaired liver function at screening
- Evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit
- Participation in a clinical study or use of an investigational compound in the 3 months preceding the first day of study drug administration, or plans to enter such a study during the study period.
- Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
About this study
Official title: An Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy and Safety of Biostate® in Subjects With Von Willebrand Disease.
The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD).
Pharmacokinetic Component:
Read more
PK parameters will be determined from a subgroup of subjects. Subjects who complete the PK component will subsequently continue in the efficacy component of the study, either continuing on a previously established prophylaxis regimen or continuing to receive on-demand treatment with the occurrence of non-surgical bleeding (NSB) events.
Efficacy Component:
Three treatment arms are defined for the efficacy component of the study. (1) Subjects who are currently being treated on a set prophylaxis regimen with a VWF product at the time of study entry will be enrolled in the "Prophylaxis" arm. (2) Subjects not being treated on a set prophylaxis regimen at the time of study entry who require a VWF product for the treatment of NSB events will be enrolled in the "On-demand" arm and commence using Biostate in the treatment of NSB events. (3) Subjects enrolled in the "On-demand" arm have the possibility to enter the "Cross-over to Prophylaxis" arm to receive an additional 12 months of prophylactic treatment.
- 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.
Biostate®Biological
Also called Human Coagulation Factor VIII / von Willebrand Factor
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
Biostate®Biological
Also called Human Coagulation Factor VIII / von Willebrand Factor
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 |
|---|---|---|
| PK | Experimental | Includes subjects participating in the pharmacokinetic component of the study. Receives: Biological: Biostate® |
| Prophylaxis | Experimental | Includes subjects receiving 12 months of prophylactic therapy. Receives: Biological: Biostate® |
| On-demand | Experimental | Includes subjects receiving 12 months of on-demand treatment. Receives: Biological: Biostate® |
| Cross-over to prophylaxis | Experimental | Includes subjects completing 12 months of on-demand treatment (the "On-demand" arm) who cross-over to prophylactic therapy for an additional 12-month period. Receives: Biological: Biostate® |
What is being measured
The primary outcome is the main question the study is designed to answer.
Haemostatic efficacy at time of non-surgical bleeding (NSB) event
Measured: From Day 1 until final study visit
Haemostatic efficacy overall
Measured: Monthly (prophylactic therapy) or once every 3 months (for on-demand use)
Number of treatments with blood product transfusions required to resolve any bleeding event
Measured: From Day 1 until final study visit
vWF/FVIII concentrate usage (number of infusions, IU/kg per dose, per event, per month and per year)
Measured: From Day 1 until final study visit
Assessment of blood loss during any surgical procedure
Measured: From Day 1 until final study visit
Number of spontaneous or traumatic NSB events
Measured: From Day 1 until final study visit
Pharmacokinetic parameters for vWF and FVIII (PK arm only)
Measured: Up to 72 hours following infusions on Day 1 and approximately Day 180
Secondary outcomes (2)
Development of FVIII inhibitors
Measured: From Day 1 until final study visit
Development of vWF inhibitors
Measured: From Day 1 until final study visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2009 (estimated)
- Study startJun 2009
- Primary completionFeb 2012
- Study completionFeb 2012
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 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Parexel
- Responsible party
- Sponsor
- Investigators
- Program Director, Clinical R&D, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00941616
- Sponsor's protocol ID
- CSLCT-BIO-08-54
- Other IDs
- 1481, 2008-004922-18, 2008
- Status last verified
- Oct 2017
- First submitted
- 15 Jul 2009
- 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 Study to Assess the Pharmacokinetics, Efficacy and Safety of Biostate® in Subjects With Von Willebrand Disease.
- ClinicalTrials.gov ID
- NCT00941616
- Acronym / sponsor ID
- CSLCT-BIO-08-54
- Conditions
- Von Willebrand Disease
- Keywords
- Von Willebrand Disease
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Collaborators
- Parexel
- Study start
- Jun 2009
- Primary completion
- Feb 2012 (actual)
- Study completion
- Feb 2012 (actual)
- First posted
- 17 Jul 2009 (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 this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD).
Pharmacokinetic Component:
PK parameters will be determined from a subgroup of subjects. Subjects who complete the PK component will subsequently continue in the efficacy component of the study, either continuing on a previously established prophylaxis regimen or continuing to receive on-demand treatment with the occurrence of non-surgical bleeding (NSB) events.
Efficacy Component:
Three treatment arms are defined for the efficacy component of the study. (1) Subjects who are currently being treated on a set prophylaxis regimen with a VWF product at the time of study entry will be enrolled in the "Prophylaxis" arm. (2) Subjects not being treated on a set prophylaxis regimen at the time of study entry who require a VWF product for the treatment of NSB events will be enrolled in the "On-demand" arm and commence using Biostate in the treatment of NSB events. (3) Subjects enrolled in the "On-demand" arm have the possibility to enter the "Cross-over to Prophylaxis" arm to receive an additional 12 months of prophylactic treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| PK | Experimental | Biological: Biostate®Includes subjects participating in the pharmacokinetic component of the study. |
| Prophylaxis | Experimental | Biological: Biostate®Includes subjects receiving 12 months of prophylactic therapy. |
| On-demand | Experimental | Biological: Biostate®Includes subjects receiving 12 months of on-demand treatment. |
| Cross-over to prophylaxis | Experimental | Biological: Biostate®Includes subjects completing 12 months of on-demand treatment (the "On-demand" arm) who cross-over to prophylactic therapy for an additional 12-month period. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Diagnosed with VWD
- Desmopressin acetate (DDAVP) treatment is ineffective or contraindicated or not available
- Evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B) within 10 years prior to their first dose of Biostate®
- Written informed consent given
Key exclusion criteria (12 listed)
- Actively bleeding immediately prior to initial PK period
- Have received DDAVP or a VWF product in the 5 days prior to their first dose of study product
- Have Type 2B, 2N or 2M VWD
- Requiring a VWF product for a planned surgical procedure at enrolment
- Have received aspirin or other non-steroidal anti-inflammatory drugs within 7 days prior to their first dose of study product
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 |
|---|---|---|
| Biostate®Also: Human Coagulation Factor VIII / von Willebrand Factor80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180 | Biological | PK |
| Biostate®Also: Human Coagulation Factor VIII / von Willebrand FactorFrequency 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 | Cross-over to prophylaxis, On-demand, Prophylaxis |
Primary outcome
Haemostatic efficacy at time of non-surgical bleeding (NSB) event
Time frame: From Day 1 until final study visit
Haemostatic efficacy overall
Time frame: Monthly (prophylactic therapy) or once every 3 months (for on-demand use)
Number of treatments with blood product transfusions required to resolve any bleeding event
Time frame: From Day 1 until final study visit
vWF/FVIII concentrate usage (number of infusions, IU/kg per dose, per event, per month and per year)
Time frame: From Day 1 until final study visit
Assessment of blood loss during any surgical procedure
Time frame: From Day 1 until final study visit
Number of spontaneous or traumatic NSB events
Time frame: From Day 1 until final study visit
Pharmacokinetic parameters for vWF and FVIII (PK arm only)
Time frame: Up to 72 hours following infusions on Day 1 and approximately Day 180
2 secondary outcomes
Development of FVIII inhibitors
Time frame: From Day 1 until final study visit
Development of vWF inhibitors
Time frame: From Day 1 until final study visit
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.
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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
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Biostate® in people with Von Willebrand Disease, run by CSL Behring at 5 sites in 4 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 4 inclusion and 12 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 5 sites in Poland, Bulgaria, 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 at time of non-surgical bleeding (NSB) event (time frame: From Day 1 until final study visit). The study lists 7 primary outcomes in all. 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.