Von Willebrand DiseasePhase 3NCT01213446
Study of Biostate® in Children With Von Willebrand Disease
A Phase 3 study testing Biostate in people with Von Willebrand Disease, run by CSL Behring at 7 sites in 6 countries.
Trial summary
This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (VWD) in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or… Read more about this study
- Who is it for?
- Ages Up to 12 years
- 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
- 17 participants
- Where
- 7 sitesin 6 countries
- Study dates
- Aug 2010to Aug 2013
- 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 Up to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male and female subjects between 0 and <12 years of age
- Diagnosed with VWD Type 1, 2A, or 3
- Desmopressin acetate (DDAVP) treatment is ineffective, contraindicated, or not available for subject
- von Willebrand factor: ristocetin cofactor (VWF:RCo) is <20% at screening or the subject has a history of VWF:RCo <10%
- Evidence of vaccination against hepatitis A and B or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunization
- Written informed consent given
Who cannot join
- Active bleeding immediately prior to initial PK period
- Received treatment with DDAVP or a VWF concentrate product for their VWD in the 5 days prior to their first study treatment
- Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of commencing the PK period.
- Known history or suspicion of having VWF or FVIII inhibitors
- 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 other FVIII/VWF concentrates
- Participation in a clinical study or use of an investigational compound in another study in the 3 months preceding study start
- Unwillingness and/or inability to comply with the study requirements
Trial locations and contacts
7 sites in 6 countries.
7 sites in 6 countries
About this study
Official title: A Phase III Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy, and Safety of Biostate® in Paediatric Subjects With Von Willebrand Disease
This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (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
PK component: Single bolus infusion of 80 IU VWF:RCo/kg administered intravenously on Day 1, and approximately Day 180 in Type 3 VWD subjects only.
Efficacy component: Repeated bolus doses over 12 months as required to manage VWD condition.
| Study group | Role | What they receive |
|---|---|---|
| Biostate | 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: From Day 1 until final study visit
Incremental Recovery of VWF
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Incremental Recovery of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Half-life of VWF
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Half-life of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Area under the concentration curve (AUC) of VWF
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
AUC of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Maximum plasma concentration (Cmax) of VWF
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Maximum plasma concentration (Cmax) of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Minimum plasma concentration (Cmin) of VWF
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Minimum plasma concentration (Cmin) of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Time to maximum concentration (tmax) of VWF
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Time to maximum concentration (tmax) of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Mean residence time (MRT) of VWF
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Mean residence time (MRT) of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Clearance (CL) of VWF
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Clearance (CL) of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Volume of distribution of steady state (Vss) of VWF
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Volume of distribution of steady state (Vss) of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Secondary outcomes (8)
Frequency of adverse events (AEs) per infusion
Measured: 13 months
Severity of AEs per infusion
Measured: 13 months
Severity of AEs per subject
Measured: 13 months
Relatedness of AEs per infusion
Measured: 13 months
Relatedness of AEs per subject
Measured: 13 months
Development of VWF inhibitors
Measured: Sample taken at baseline, then every 3 months up to 12 months
Development of FVIII inhibitors
Measured: Sample taken at baseline, then every 3 months up to 12 months
Frequency of adverse events (AEs) per subject
Measured: 13 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2010 (estimated)
- Study startAug 2010
- Primary completionAug 2013
- Study completionAug 2013
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
- 17 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
- NCT01213446
- Sponsor's protocol ID
- CSLCT-BIO-08-52
- Other IDs
- 2009-017753-34, 1494, 2009
- Status last verified
- Oct 2017
- First submitted
- 1 Oct 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
- A Phase III Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy, and Safety of Biostate® in Paediatric Subjects With Von Willebrand Disease
- ClinicalTrials.gov ID
- NCT01213446
- Acronym / sponsor ID
- CSLCT-BIO-08-52
- Conditions
- Von Willebrand Disease
- Keywords
- Von Willebrand Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Collaborators
- Parexel
- Study start
- Aug 2010
- Primary completion
- Aug 2013 (actual)
- Study completion
- Aug 2013 (actual)
- First posted
- 4 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.
This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (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 |
|---|---|---|
| Biostate | Experimental | Biological: Biostate |
Who may qualify
- Ages
- Up to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Male and female subjects between 0 and <12 years of age
- Diagnosed with VWD Type 1, 2A, or 3
- Desmopressin acetate (DDAVP) treatment is ineffective, contraindicated, or not available for subject
- von Willebrand factor: ristocetin cofactor (VWF:RCo) is <20% at screening or the subject has a history of VWF:RCo <10%
- Evidence of vaccination against hepatitis A and B or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunization
Key exclusion criteria (8 listed)
- Active bleeding immediately prior to initial PK period
- Received treatment with DDAVP or a VWF concentrate product for their VWD in the 5 days prior to their first study treatment
- Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of commencing the PK period.
- Known history or suspicion of having VWF or FVIII inhibitors
- Acute or chronic medical condition, other than VWD, which may affect the conduct of the study
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 |
|---|---|---|
| BiostatePK component: Single bolus infusion of 80 IU VWF:RCo/kg administered intravenously on Day 1, and approximately Day 180 in Type 3 VWD subjects only. Efficacy component: Repeated bolus doses over 12 months as required to manage VWD condition. | Biological | Biostate |
Primary outcome
Haemostatic efficacy
Time frame: From Day 1 until final study visit
Incremental Recovery of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Incremental Recovery of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Half-life of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Half-life of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Area under the concentration curve (AUC) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
AUC of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Maximum plasma concentration (Cmax) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Maximum plasma concentration (Cmax) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Minimum plasma concentration (Cmin) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Minimum plasma concentration (Cmin) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Time to maximum concentration (tmax) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Time to maximum concentration (tmax) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Mean residence time (MRT) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Mean residence time (MRT) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Clearance (CL) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Clearance (CL) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Volume of distribution of steady state (Vss) of VWF
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Volume of distribution of steady state (Vss) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
8 secondary outcomes
Frequency of adverse events (AEs) per infusion
Time frame: 13 months
Severity of AEs per infusion
Time frame: 13 months
Severity of AEs per subject
Time frame: 13 months
Relatedness of AEs per infusion
Time frame: 13 months
Relatedness of AEs per subject
Time frame: 13 months
Development of VWF inhibitors
Time frame: Sample taken at baseline, then every 3 months up to 12 months
Development of FVIII inhibitors
Time frame: Sample taken at baseline, then every 3 months up to 12 months
Frequency of adverse events (AEs) per subject
Time frame: 13 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.
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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
7 sites in 6 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 7 sites in 6 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 6 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 7 sites in Belarus, Georgia, Germany, Guatemala, Lebanon and 1 more country. 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: From Day 1 until final study visit). The study lists 19 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.