Register of Patients With haEmophilia A tReated With Afstyla® (OPERA)

CSL Behring

Status: Completed

Conditions: Haemophilia A

Treatments: Biological: Afstyla®

Study type: Observational

Funder types: Industry

Identifiers

NCT04675541

2017-A03517-46 (Other Identifier)

OPERA study

Details and patient eligibility

About

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

Full 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.

Enrollment: 62 patients

Sex: All

Volunteers: No Healthy Volunteers

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.