Brief Title
Register of Patients With haEmophilia A tReated With Afstyla®
Official Title
Observational Register of Patients With haEmophilia A tReated With Afstyla®
Brief Summary
Record real life data of patients with Hemophilia A and treated with Afstyla® to assess the effectiveness and the safety of the treatment used as prophylaxis, prevention of bleeding (e.g. surgery) or on-demand treatment during 3 years after patient inclusion
Detailed Description
Haemophilia A is a congenital coagulation disorder caused by a deficiency or abnormality of coagulation factor VIII (FVIII). The severity of the haemophilia depends on the magnitude of the FVIII deficiency and the clinical features depend on the site of the bleed. The therapeutic management of this disease involves administering the deficient factor, FVIII to the patient. Depending on the severity of the disease and patient background, the management regimens are different (long term prophylactic treatment to prevent non surgical bleeds, prophylactic short term treatment to prevent surgical bleeds or treatment of acute bleeds on demand). Several recombinant or plasma derived treatments are currently available. The CSL Behring Company has designed a new recombinant single chain B1FVIII, rFVIII (Afstyla®). This has increased affinity for VWF, resulting in improved stability and an improvement in its pharmacokinetic indices. OPERA is a non-interventional, prospective and national study which aim is to record real life data in haemophilia A French patients treated with Afstyla® in order to confirm the efficacy and safety of this proprietary product established in clinical development studies.
Study Type
Observational
Primary Outcome
The annualized bleeding rate (spontaneous, traumatic) during long term prophylactic and on demand regimen.
Secondary Outcome
The number of infusions of Afstyla® required during the surgical procedures
Condition
Haemophilia A
Intervention
Afstyla®
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
62
Start Date
September 24, 2018
Completion Date
December 2024
Primary Completion Date
December 2024
Eligibility Criteria
Inclusion Criteria: - Have given their agreement to take part in the observational registry after being informed in writing of the purposes of the study and after their data have been recorded (parent's agreement for minor patients); - Be suffering from haemophilia A and being treated or having been treated with Afstyla® long term prophylactically, on demand or preventatively for a surgical procedure; - Absence of inhibitor and/or treatment for immune tolerance at the time of inclusion 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 Afstyla® treatment (known hypersensitivity to FVIII or hamster proteins); - Simultaneous participation in an interventional clinical study. - Presence of an inhibitor and/or ongoing immune tolerance.
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Study Director, ,
Location Countries
France
Location Countries
France
Administrative Informations
NCT ID
NCT04675541
Organization ID
OPERA study
Secondary IDs
2017-A03517-46
Responsible Party
Sponsor
Study Sponsor
CSL Behring
Study Sponsor
Study Director, Study Director, CSL Behring SA
Verification Date
January 2022