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Registry of Patients With Von WilLEbrand Disease Treated With Voncento® (OPALE)

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ClinicalTrials.gov Identifier: NCT04657887
Recruitment Status : Active, not recruiting
First Posted : December 8, 2020
Last Update Posted : January 11, 2022
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Study Description
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Brief 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.

Condition or disease Intervention/treatment
Von Willebrand Disease Biological: Voncento®

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
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Study Type : Observational
Actual Enrollment : 135 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Observational Registry of Patients With Von WilLEbrand Disease Treated With Voncento®
Actual Study Start Date : November 23, 2015
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2023

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Intervention Details:
  • Biological: Voncento®
    Solution for injection
    Other Name: Biostate

Outcome Measures
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Primary Outcome Measures :
  1. 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 ]
  2. Number of non-surgical bleeding episodes per year [ Time Frame: Up to 24 months ]
  3. 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 ]
  4. 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 ]

Secondary Outcome Measures :
  1. 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 ]
  2. Number of administrations of Voncento® needed to prevent or treat surgical bleeding episode [ Time Frame: Up to 24 months ]
  3. Total dose of Voncento® (in IU/kg of VWF) needed to prevent or treat surgical bleeding episode [ Time Frame: Up to 24 months ]
  4. Nature and impact of adverse events and in particular serious adverse events, adverse events related to Voncento® [ Time Frame: Up to 24 months ]
  5. Collection of available biological data (ex: FVIII, VWF:Rco, VWF:Ag) [ Time Frame: At baseline and up to 24 months ]

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
French cohort of patients with inherited von Willebrand disease treated with Voncento.
Criteria

Inclusion Criteria:

  • 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)

Exclusion Criteria:

  • 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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04657887


Locations
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Sponsors and Collaborators
CSL Behring
Investigators
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Study Director: Study Director CSL Behring SA
More Information
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Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT04657887    
Other Study ID Numbers: OPALE study
First Posted: December 8, 2020    Key Record Dates
Last Update Posted: January 11, 2022
Last Verified: January 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by CSL Behring:
vWD
von Willebrand Factor
vWF
Additional relevant MeSH terms:
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Von Willebrand Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn