Registry of Patients With Von WilLEbrand Disease Treated With Voncento®

Von Willebrand Disease Clinical Trial

— OPALE  

Official title:

Observational Registry of Patients With Von WilLEbrand Disease Treated With Voncento®

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04657887
• Other study ID #: OPALE study
• Status: Completed
• Start date: November 23, 2015
• Est. completion date: December 31, 2023

Study information

• Verified date: April 2024
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 135
• Est. completion date: December 31, 2023
• Est. primary completion date: December 31, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Von Willebrand Disease
• Von Willebrand Diseases

Intervention

Biological:
• Voncento®
Solution for injection

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Number of non-surgical bleeding episodes per year		Up to 24 months
Primary	Number of administrations of Voncento® needed to treat a non-surgical bleeding episode and for the long term prophylaxis		Up to 24 months
Primary	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	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
Secondary	Number of administrations of Voncento® needed to prevent or treat surgical bleeding episode		Up to 24 months
Secondary	Total dose of Voncento® (in IU/kg of VWF) needed to prevent or treat surgical bleeding episode		Up to 24 months
Secondary	Nature and impact of adverse events and in particular serious adverse events, adverse events related to Voncento®		Up to 24 months
Secondary	Collection of available biological data (ex: FVIII, VWF:Rco, VWF:Ag)		At baseline and up to 24 months