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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04657887
Other study ID # OPALE study
Secondary ID
Status Completed
Phase
First received
Last updated
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
Health authority
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

Study Design


Related Conditions & MeSH terms


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
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