Von Willebrand DiseasePhase 4NCT02552576
Study of Voncento® in Subjects With Von Willebrand Disease
A Phase 4 study testing Voncento in people with Von Willebrand Disease, run by CSL Behring at 12 sites in 5 countries.
Trial summary
This is a multi-centre, open-label, single-arm, phase 4, post-marketing study to further investigate the efficacy and safety of Voncento in subjects with Von Willebrand Disease (VWD) in whom treatment with a Von Willebrand Factor (VWF) product is required as on-demand therapy, for prophylactic therapy, or during… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Von Willebrand Disease
- What is being tested?
- Voncento
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 12 sitesin 5 countries
- Study dates
- 5 Oct 2015to 15 Feb 2018
- Phase
- Phase 4
- 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
- Diagnosis of severe type 1, 2A, or 3 VWD where Von Willebrand Factor: Ristocetin Cofactor (VWF:RCo) is <20% at screening
- Desmopressin acetate treatment is ineffective, contraindicated, or not available for subject (type 3 VWD subjects only).
- 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.
- Require a VWF product to control a non-surgical bleeding (NSB) event or for ongoing prophylactic therapy.
Who cannot join
- 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 Voncento, VWF / FVIII concentrates, or human albumin.
- Participated in another interventional clinical study within 30 days before the first administration of Voncento or at any time during the study.
- Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening
- Alcohol, drug, or medication abuse within 1 year before the study.
- Currently receiving a therapy not permitted during the study.
- Previous participation in a Voncento / Biostate study.
Trial locations and contacts
12 sites in 5 countries.
12 sites in 5 countries
United Kingdom5 sites
Study Site 14
London
Study Site 40
London
Study Site 42
London
Study Site 47
London
Study Site 8
London
About this study
Official title: An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects With Von Willebrand Disease
This is a multi-centre, open-label, single-arm, phase 4, post-marketing study to further investigate the efficacy and safety of Voncento in subjects with Von Willebrand Disease (VWD) in whom treatment with a Von Willebrand Factor (VWF) product is required as on-demand therapy, for prophylactic therapy, or during surgery. Subjects will be treated with Voncento either as an on-demand regimen (eg, to treat a non-surgical spontaneous or traumatic bleeding event) or prevention regimen (eg, to prevent an anticipated bleeding event) at a dose prescribed by the Investigator in accordance with the Voncento Summary of Product Characteristics (SmPC), or with a prophylaxis regimen (regular treatment with Voncento at a frequency of 1-3 times per week). Voncento will also be given to prevent and treat any surgical bleeding events.
- Conditions
- Von Willebrand Disease
- Being tested
- Voncento
- 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.
VoncentoBiological
Human coagulation VWF / coagulation factor VIII (FVIII) complex concentrate
| Study group | Role | What they receive |
|---|---|---|
| Voncento | Experimental | The frequency and dose of Voncento administration will be determined by the investigator using the information included in the Voncento Summary of product characteristics (SmPC) Receives: Biological: Voncento |
What is being measured
The primary outcome is the main question the study is designed to answer.
Haemostatic efficacy - NSB event
Subject's and investigator's assessment of haemostatic efficacy of Voncento in its usage for a non-surgical bleeding (NSB) event. Assessments of haemostatic efficacy will be based on a 4-point ordinal scale of Excellent, Good, Moderate or None.
Measured: Assessed daily by the subject until the bleed stops, for the duration of the subject's participation in the study (approximately 12 months). Each bleeding event is also to be assessed retrospectively by the Investigator.
Number of infusions - NSB event
Number of infusions of Voncento required to treat an NSB event.
Measured: For the duration of the subject's participation in the study (approximately 12 months).
Total dose of Voncento - NSB event
Total dose of Voncento (in international units of Von Willebrand Factor: Ristocetin Cofactor) required to treat an NSB event.
Measured: For the duration of the subject's participation in the study (approximately 12 months).
Number of NSB events per month
Measured: From Day 1 until final study visit, approximately 12 months.
Annual bleeding rate
The number of bleeding events per year
Measured: For the duration of the subject's participation in the study (approximately 12 months).
Secondary outcomes (7)
Assessment of blood loss during a surgical procedure
Measured: During surgery, for any surgical procedure during the subject's participation in the study (approximately 12 months).
Haemostatic efficacy - surgical event
Investigator's or surgeon's assessment of haemostatic efficacy of Voncento in its usage for a surgical bleeding event. Assessments of haemostatic efficacy will be based on a 4-point ordinal scale of Excellent, Good, Moderate or None.
Measured: Assessed during and after surgery until the bleeding stops, for any surgical procedure during the subject's participation in the study (approximately 12 months).
Number of infusions - surgical bleeding event
Number of infusions of Voncento required to treat a surgical bleeding event.
Measured: For the duration of the subject's participation in the study (approximately 12 months).
Total dose of Voncento - surgical bleeding event
Total dose of Voncento (in international units of Von Willebrand Factor: Ristocetin Cofactor) required to treat a surgical bleeding event.
Measured: For the duration of the subject's participation in the study (approximately 12 months).
Overall adverse events
Overall number of subjects with: adverse events (AEs), serious AEs, AEs related to Voncento administration, and adverse events of special interest.
Measured: From Day 1 until the final study visit for each subject (approximately 12 months)
Number of subjects with VWF or FVIII inhibitors
Measured: At screening, Day 1 and approximately Months 3, 6, 9 and 12, for each subject.
Haemostatic efficacy - prophylaxis
Subject's and investigator's assessment of haemostatic efficacy of Voncento as prophylaxis therapy. Assessments of haemostatic efficacy will be based on a grading scale with outcomes of excellent, good, moderate, none.
Measured: Approximately every month (subject assessment) and every 3 months (Investigator assessment) for the duration of the subject's participation in the study (approximately 12 months).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2015 (estimated)
- Study start5 Oct 2015
- Primary completion15 Feb 2018
- Study completion15 Feb 2018
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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02552576
- Sponsor's protocol ID
- CSLCT-BIO-12-83
- Other IDs
- 2013-003305-25
- Status last verified
- May 2018
- First submitted
- 15 Sep 2015
- Last updated
- 16 May 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects With Von Willebrand Disease
- ClinicalTrials.gov ID
- NCT02552576
- Acronym / sponsor ID
- CSLCT-BIO-12-83
- Conditions
- Von Willebrand Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- 5 Oct 2015 (actual)
- Primary completion
- 15 Feb 2018 (actual)
- Study completion
- 15 Feb 2018 (actual)
- First posted
- 17 Sep 2015 (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 a multi-centre, open-label, single-arm, phase 4, post-marketing study to further investigate the efficacy and safety of Voncento in subjects with Von Willebrand Disease (VWD) in whom treatment with a Von Willebrand Factor (VWF) product is required as on-demand therapy, for prophylactic therapy, or during surgery. Subjects will be treated with Voncento either as an on-demand regimen (eg, to treat a non-surgical spontaneous or traumatic bleeding event) or prevention regimen (eg, to prevent an anticipated bleeding event) at a dose prescribed by the Investigator in accordance with the Voncento Summary of Product Characteristics (SmPC), or with a prophylaxis regimen (regular treatment with Voncento at a frequency of 1-3 times per week). Voncento will also be given to prevent and treat any surgical bleeding events.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Voncento | Experimental | Biological: VoncentoThe frequency and dose of Voncento administration will be determined by the investigator using the information included in the Voncento Summary of product characteristics (SmPC) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of severe type 1, 2A, or 3 VWD where Von Willebrand Factor: Ristocetin Cofactor (VWF:RCo) is <20% at screening
- Desmopressin acetate treatment is ineffective, contraindicated, or not available for subject (type 3 VWD subjects only).
- 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.
- Require a VWF product to control a non-surgical bleeding (NSB) event or for ongoing prophylactic therapy.
Key exclusion criteria (8 listed)
- 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 Voncento, VWF / FVIII concentrates, or human albumin.
- Participated in another interventional clinical study within 30 days before the first administration of Voncento or at any time during the study.
- Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening
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 |
|---|---|---|
| VoncentoHuman coagulation VWF / coagulation factor VIII (FVIII) complex concentrate | Biological | Voncento |
Primary outcome
Haemostatic efficacy - NSB event
Time frame: Assessed daily by the subject until the bleed stops, for the duration of the subject's participation in the study (approximately 12 months). Each bleeding event is also to be assessed retrospectively by the Investigator.
Subject's and investigator's assessment of haemostatic efficacy of Voncento in its usage for a non-surgical bleeding (NSB) event. Assessments of haemostatic efficacy will be based on a 4-point ordinal scale of Excellent, Good, Moderate or None.
Number of infusions - NSB event
Time frame: For the duration of the subject's participation in the study (approximately 12 months).
Number of infusions of Voncento required to treat an NSB event.
Total dose of Voncento - NSB event
Time frame: For the duration of the subject's participation in the study (approximately 12 months).
Total dose of Voncento (in international units of Von Willebrand Factor: Ristocetin Cofactor) required to treat an NSB event.
Number of NSB events per month
Time frame: From Day 1 until final study visit, approximately 12 months.
Annual bleeding rate
Time frame: For the duration of the subject's participation in the study (approximately 12 months).
The number of bleeding events per year
7 secondary outcomes
Assessment of blood loss during a surgical procedure
Time frame: During surgery, for any surgical procedure during the subject's participation in the study (approximately 12 months).
Haemostatic efficacy - surgical event
Time frame: Assessed during and after surgery until the bleeding stops, for any surgical procedure during the subject's participation in the study (approximately 12 months).
Number of infusions - surgical bleeding event
Time frame: For the duration of the subject's participation in the study (approximately 12 months).
Total dose of Voncento - surgical bleeding event
Time frame: For the duration of the subject's participation in the study (approximately 12 months).
Overall adverse events
Time frame: From Day 1 until the final study visit for each subject (approximately 12 months)
Number of subjects with VWF or FVIII inhibitors
Time frame: At screening, Day 1 and approximately Months 3, 6, 9 and 12, for each subject.
Haemostatic efficacy - prophylaxis
Time frame: Approximately every month (subject assessment) and every 3 months (Investigator assessment) for the duration of the subject's participation in the study (approximately 12 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 16 May 2018.
Related trials for Von Willebrand Disease
Active, not recruitingObservational study
An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
Von Willebrand Disease, Type 3
RecruitingObservational study
Heavy Menstrual Bleeding Progestin Treatment in Bleeding Disorders Study
Heavy Menstrual Bleeding, Bleeding Disorder
RecruitingPhase 2
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB)
RecruitingObservational study
Blood Clotting Markers and Heart Ultrasound in People With Device-Detected Atrial Fibrillation
Device-detected Atrial Fibrillation, Subclinical Atrial Fibrillation
RecruitingPhase 3
A Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients With VWD
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.
Study Director study director
CSL Behring
12 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Voncento in people with Von Willebrand Disease, run by CSL Behring at 12 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 5 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 16 May 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United Kingdom, Poland, Germany, Austria, Greece. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 - NSB event (time frame: Assessed daily by the subject until the bleed stops, for the duration of the subject's participation in the study (approximately 12 months). Each bleeding event is also to be assessed retrospectively by the Investigator.). The study lists 5 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 16 May 2018.