Von Willebrand DiseaseOPALENCT04657887
Registry of Patients With Von WilLEbrand Disease Treated With Voncento®
An observational study of Von Willebrand Disease, run by CSL Behring at 17 sites in France.
Trial summary
Description of the long-term evolution of patients with von Willebrand disease and treated with Voncento® and of the hemostatic efficacy in the prevention and the treatment of non-surgical bleeding episodes and prevention of surgical bleeding during 2 years after patient inclusion. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Von Willebrand Disease
- What is being tested?
- Voncento®
- Type of treatment
- Injection or infusion
- How many people
- 135 participants
- Where
- 17 sitesFrance
- Study dates
- 23 Nov 2015to 31 Dec 2023
- Phase
- Observational
- 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
Study population: French cohort of patients with inherited von Willebrand disease treated with Voncento.
Who may be able to join
- Patients suffering from constitutional von Willebrand disease for whom Desmopressin treatment is deemed ineffective or contraindicated
- Patients suffering from von Willebrand disease and being treated or having been treated with Voncento® for the treatment of surgical and non-surgical bleeding episodes, the prophylaxis of surgical and non-surgical bleedings
- Patients with no history or suspicion of inhibitors (judged on previous efficacy)
Who cannot join
- Refusal of the patient or the patient's legal representative to take part in the study;
- Existence of a contraindication to the use of Voncento® treatment
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
France17 sites
CHU Besançon
Besançon
CHU Bordeaux
Bordeaux
CHU Morvan Brest
Brest
CHU Lyon
Bron
CHU Caen
Caen
Hôpital Simone Veil
Eaubonne
Hôpital Mignot
Le Chesnay
Hôpital Bicêtre
Le Kremlin-Bicêtre
Hôpital Saint-Eloi
Montpellier
CHRU Nancy
Nancy
CHU Nantes
Nantes
CHU Lariboisière
Paris
Hôpital Cochin
Paris
Hôpital Necker
Paris
CHU Rennes
Rennes
CHU Rouen
Rouen
CHU Strasbourg
Strasbourg
About this study
Official title: Observational Registry of Patients With Von WilLEbrand Disease Treated With Voncento®
Description of the long-term evolution of patients with von Willebrand disease and treated with Voncento® and of the hemostatic efficacy in the prevention and the treatment of non-surgical bleeding episodes and prevention of surgical bleeding during 2 years after patient inclusion.
Read the detailed description
Inherited von Willebrand disease (VWD) is considered the most common bleeding disorder. Its prevalence is approximately 1% in the general population but symptomatic patients are rarer (0.01%). It is caused by a partial or total quantitative deficiency (type 1 and type 3) or by a qualitative defect (type 2) of von Willebrand factor (VWF), a large multimeric protein that is required for platelet adhesion and serves as factor VIII (FVIII) carrier. Type 2 VWD is further divided in four subgroups (2A, 2B, 2M, and 2N) that are distinguished according to the nature of the VWF defect. Most patients with type 1 VWD can be treated with the synthetic vasopressin analogue desmopressin (DDAVP; 2-desamino-8-D-arginine vasopressin), whereas patients with type 3 VWD and most patients with type 2 VWD require concentrates containing VWF. Plasma-derived FVIII concentrates, which were initially developed for the treatment of haemophilia, contain large amounts of VWF and are used in patients for whom DDAVP treatment is deemed ineffective or contraindicated. Voncento® (CSL Behring) is a plasma-derived FVIII/VWF concentrate registered in France since 2015 for the treatment and prevention of bleeding events in patients with inherited VWD. OPALE is an observational study describing the use of human coagulation FVIII/VWF concentrate (Voncento®) to treat and prevent bleeding episodes in a French cohort of patients with inherited von Willebrand disease in the real life settings. The aim of the OPALE study is to describe the efficacy and the safety of Voncento® in the prophylaxis and treatment of haemorrhage or surgical bleeding.
- Conditions
- Von Willebrand Disease
- Being tested
- Voncento®
- Keywords
- vWD, von Willebrand Factor, vWF
- 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.
Voncento®Biological
Also called Biostate
Solution for injection
What is being measured
The primary outcome is the main question the study is designed to answer.
Global assessment by the patient and the investigator of the hemostatic efficacy of Voncento® in the management of non-surgical bleeding episodes
Measured: Up to 24 months
Number of non-surgical bleeding episodes per year
Measured: Up to 24 months
Number of administrations of Voncento® needed to treat a non-surgical bleeding episode and for the long term prophylaxis
Measured: Up to 24 months
Total dose of Voncento® (in IU/kg of VWF) needed to treat a non-surgical bleeding episode and for the long term prophylaxis
Measured: Up to 24 months
Secondary outcomes (5)
Assessment by the investigator of the hemostatic efficacy of Voncento® during the treatment and the prophylaxis of surgical bleedings and after surgical procedures
Measured: Up to 24 months
Number of administrations of Voncento® needed to prevent or treat surgical bleeding episode
Measured: Up to 24 months
Total dose of Voncento® (in IU/kg of VWF) needed to prevent or treat surgical bleeding episode
Measured: Up to 24 months
Nature and impact of adverse events and in particular serious adverse events, adverse events related to Voncento®
Measured: Up to 24 months
Collection of available biological data (ex: FVIII, VWF:Rco, VWF:Ag)
Measured: At baseline and up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2020
- Study start23 Nov 2015
- Primary completion31 Dec 2023
- Study completion31 Dec 2023
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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 135 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 SA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04657887
- Sponsor's protocol ID
- OPALE study
- Acronym
- OPALE
- Status last verified
- Apr 2024
- First submitted
- 1 Dec 2020
- Last updated
- 19 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Registry of Patients With Von WilLEbrand Disease Treated With Voncento®
- ClinicalTrials.gov ID
- NCT04657887
- Acronym / sponsor ID
- OPALE / OPALE study
- Conditions
- Von Willebrand Disease
- Keywords
- vWD, von Willebrand Factor, vWF
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 135 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- 23 Nov 2015 (actual)
- Primary completion
- 31 Dec 2023 (actual)
- Study completion
- 31 Dec 2023 (actual)
- First posted
- 8 Dec 2020 (actual)
- 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.
Description of the long-term evolution of patients with von Willebrand disease and treated with Voncento® and of the hemostatic efficacy in the prevention and the treatment of non-surgical bleeding episodes and prevention of surgical bleeding during 2 years after patient inclusion.
Detailed description
Inherited von Willebrand disease (VWD) is considered the most common bleeding disorder. Its prevalence is approximately 1% in the general population but symptomatic patients are rarer (0.01%). It is caused by a partial or total quantitative deficiency (type 1 and type 3) or by a qualitative defect (type 2) of von Willebrand factor (VWF), a large multimeric protein that is required for platelet adhesion and serves as factor VIII (FVIII) carrier. Type 2 VWD is further divided in four subgroups (2A, 2B, 2M, and 2N) that are distinguished according to the nature of the VWF defect. Most patients with type 1 VWD can be treated with the synthetic vasopressin analogue desmopressin (DDAVP; 2-desamino-8-D-arginine vasopressin), whereas patients with type 3 VWD and most patients with type 2 VWD require concentrates containing VWF. Plasma-derived FVIII concentrates, which were initially developed for the treatment of haemophilia, contain large amounts of VWF and are used in patients for whom DDAVP treatment is deemed ineffective or contraindicated. Voncento® (CSL Behring) is a plasma-derived FVIII/VWF concentrate registered in France since 2015 for the treatment and prevention of bleeding events in patients with inherited VWD. OPALE is an observational study describing the use of human coagulation FVIII/VWF concentrate (Voncento®) to treat and prevent bleeding episodes in a French cohort of patients with inherited von Willebrand disease in the real life settings. The aim of the OPALE study is to describe the efficacy and the safety of Voncento® in the prophylaxis and treatment of haemorrhage or surgical bleeding.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- French cohort of patients with inherited von Willebrand disease treated with Voncento.
Key inclusion criteria (3 listed)
- Patients suffering from constitutional von Willebrand disease for whom Desmopressin treatment is deemed ineffective or contraindicated
- Patients suffering from von Willebrand disease and being treated or having been treated with Voncento® for the treatment of surgical and non-surgical bleeding episodes, the prophylaxis of surgical and non-surgical bleedings
- Patients with no history or suspicion of inhibitors (judged on previous efficacy)
Key exclusion criteria (2 listed)
- Refusal of the patient or the patient's legal representative to take part in the study;
- Existence of a contraindication to the use of Voncento® treatment
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 |
|---|---|---|
| Voncento®Also: BiostateSolution for injection | Biological | – |
Primary outcome
Global assessment by the patient and the investigator of the hemostatic efficacy of Voncento® in the management of non-surgical bleeding episodes
Time frame: Up to 24 months
Number of non-surgical bleeding episodes per year
Time frame: Up to 24 months
Number of administrations of Voncento® needed to treat a non-surgical bleeding episode and for the long term prophylaxis
Time frame: Up to 24 months
Total dose of Voncento® (in IU/kg of VWF) needed to treat a non-surgical bleeding episode and for the long term prophylaxis
Time frame: Up to 24 months
5 secondary outcomes
Assessment by the investigator of the hemostatic efficacy of Voncento® during the treatment and the prophylaxis of surgical bleedings and after surgical procedures
Time frame: Up to 24 months
Number of administrations of Voncento® needed to prevent or treat surgical bleeding episode
Time frame: Up to 24 months
Total dose of Voncento® (in IU/kg of VWF) needed to prevent or treat surgical bleeding episode
Time frame: Up to 24 months
Nature and impact of adverse events and in particular serious adverse events, adverse events related to Voncento®
Time frame: Up to 24 months
Collection of available biological data (ex: FVIII, VWF:Rco, VWF:Ag)
Time frame: At baseline and up to 24 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 19 Apr 2024.
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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.
Study Director study director
CSL Behring SA
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand Disease, run by CSL Behring at 17 sites in France.
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 3 inclusion and 2 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 19 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in France. 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.
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?
Global assessment by the patient and the investigator of the hemostatic efficacy of Voncento® in the management of non-surgical bleeding episodes (time frame: Up to 24 months). The study lists 4 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 19 Apr 2024.