Clinical trial NCT04657887: Registry of Patients With Von WilLEbrand Disease Treated With Voncento®
Official title: Observational Registry of Patients With Von WilLEbrand Disease Treated With Voncento®
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Von Willebrand Disease
Keywords: vWD
von Willebrand Factor
vWF
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2020-12-08
Enrollment: 135
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: French cohort of patients with inherited von Willebrand disease treated with Voncento.

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.

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.

Eligibility 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

Primary outcomes:
measure: Global assessment by the patient and the investigator of the hemostatic efficacy of Voncento® in the management of non-surgical bleeding episodes
timeFrame: Up to 24 months
measure: Number of non-surgical bleeding episodes per year
timeFrame: Up to 24 months
measure: Number of administrations of Voncento® needed to treat a non-surgical bleeding episode and for the long term prophylaxis
timeFrame: Up to 24 months
measure: Total dose of Voncento® (in IU/kg of VWF) needed to treat a non-surgical bleeding episode and for the long term prophylaxis
timeFrame: Up to 24 months
Secondary outcomes:
measure: Assessment by the investigator of the hemostatic efficacy of Voncento® during the treatment and the prophylaxis of surgical bleedings and after surgical procedures
timeFrame: Up to 24 months
measure: Number of administrations of Voncento® needed to prevent or treat surgical bleeding episode
timeFrame: Up to 24 months
measure: Total dose of Voncento® (in IU/kg of VWF) needed to prevent or treat surgical bleeding episode
timeFrame: Up to 24 months
measure: Nature and impact of adverse events and in particular serious adverse events, adverse events related to Voncento®
timeFrame: Up to 24 months
measure: Collection of available biological data (ex: FVIII, VWF:Rco, VWF:Ag)
timeFrame: At baseline and up to 24 months
Other outcomes:
Locations:
city: Besançon
country: France
facility: CHU Besançon
city: Bordeaux
country: France
facility: CHU Bordeaux
city: Brest
country: France
facility: CHU Morvan Brest
city: Bron
country: France
facility: CHU Lyon
city: Caen
country: France
facility: CHU Caen
city: Eaubonne
country: France
facility: Hôpital Simone Veil
city: Le Chesnay
country: France
facility: Hôpital Mignot
city: Le Kremlin-Bicêtre
country: France
facility: Hôpital Bicêtre
city: Montpellier
country: France
facility: Hôpital Saint-Eloi
city: Nancy
country: France
facility: CHRU Nancy
city: Nantes
country: France
facility: CHU Nantes
city: Paris
country: France
facility: CHU Lariboisière
city: Paris
country: France
facility: Hôpital Cochin
city: Paris
country: France
facility: Hôpital Necker
city: Rennes
country: France
facility: CHU Rennes
city: Rouen
country: France
facility: CHU Rouen
city: Strasbourg
country: France
facility: CHU Strasbourg
Study officials:
affiliation: CSL Behring SA
name: Study Director
role: STUDY_DIRECTOR
References: