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

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.

Study Overview

• Status: Completed
• Conditions: Von Willebrand Disease
• Intervention / Treatment: 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 Type: Observational
• Enrollment (Actual): 135

Contacts and Locations

Study Locations

• France
  • Besançon, France
    • CHU Besançon
  • Bordeaux, France
    • CHU Bordeaux
  • Brest, France
    • CHU Morvan Brest
  • Bron, France
    • CHU Lyon
  • Caen, France
    • CHU Caen
  • Eaubonne, France
    • Hôpital Simone Veil
  • Le Chesnay, France
    • Hôpital Mignot
  • Le Kremlin-Bicêtre, France
    • Hôpital Bicêtre
  • Montpellier, France
    • Hôpital Saint-Eloi
  • Nancy, France
    • CHRU Nancy
  • Nantes, France
    • CHU Nantes
  • Paris, France
    • Hôpital Cochin
  • Paris, France
    • Hôpital Necker
  • Paris, France
    • CHU Lariboisière
  • Rennes, France
    • CHU Rennes
  • Rouen, France
    • CHU Rouen
  • Strasbourg, France
    • CHU Strasbourg

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

French cohort of patients with inherited von Willebrand disease treated with Voncento.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Global assessment by the patient and the investigator of the hemostatic efficacy of Voncento® in the management of non-surgical bleeding episodes	Up to 24 months
Number of non-surgical bleeding episodes per year	Up to 24 months
Number of administrations of Voncento® needed to treat a non-surgical bleeding episode and for the long term prophylaxis	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	Up to 24 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Assessment by the investigator of the hemostatic efficacy of Voncento® during the treatment and the prophylaxis of surgical bleedings and after surgical procedures	Up to 24 months
Number of administrations of Voncento® needed to prevent or treat surgical bleeding episode	Up to 24 months
Total dose of Voncento® (in IU/kg of VWF) needed to prevent or treat surgical bleeding episode	Up to 24 months
Nature and impact of adverse events and in particular serious adverse events, adverse events related to Voncento®	Up to 24 months
Collection of available biological data (ex: FVIII, VWF:Rco, VWF:Ag)	At baseline and up to 24 months

Collaborators and Investigators

• Sponsor: CSL Behring
• Investigators: Study Director: Study Director, CSL Behring SA

Study record dates

Study Major Dates

• Study Start (Actual): November 23, 2015
• Primary Completion (Actual): December 31, 2023
• Study Completion (Actual): December 31, 2023

Study Registration Dates

• First Submitted: December 1, 2020
• First Submitted That Met QC Criteria: December 7, 2020
• First Posted (Actual): December 8, 2020

Study Record Updates

• Last Update Posted (Actual): April 19, 2024
• Last Update Submitted That Met QC Criteria: April 18, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: von Willebrand Factor; vWD; vWF
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Blood Platelet Disorders; Von Willebrand Diseases
• Other Study ID Numbers: OPALE study

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No