Register of Patients With haEmophilia A tReated With Afstyla®

Haemophilia A Clinical Trial

— OPERA  

Official title:

Observational Register of Patients With haEmophilia A tReated With Afstyla®

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04675541
• Other study ID #: OPERA study
• Secondary ID: 2017-A03517-46
• Status: Active, not recruiting
• Start date: September 24, 2018
• Est. completion date: December 2024

Study information

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

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 62
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Haemophilia A
• Hemophilia A

Intervention

Biological:
• Afstyla®
Solution for injection

Locations

Country	Name	City	State
France	CHU Brest	Brest
France	CHU Caen	Caen
France	CHU Chambéry	Chambéry
France	CHU Clermont-Ferrand	Clermont-Ferrand
France	Hôpital Simone Veil	Eaubonne
France	Hôpital Mignot	Le Chesnay
France	CRC-MHC Hôpital Kremlin Bicêtre	Le Kremlin-Bicêtre
France	CHRU Lille	Lille
France	CHU Limoges	Limoges
France	CHU Montpellier	Montpellier
France	CHU Nantes	Nantes
France	Hôpital Necker	Paris
France	CHU Rennes	Rennes
France	CHU Rouen	Rouen
France	CHU Saint-Etienne	Saint-Étienne
France	Hôpital Hautepierre	Strasbourg
France	CHRU de Tours	Tours

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The annualized bleeding rate (spontaneous, traumatic) during long term prophylactic and on demand regimen.		Up to 36 months
Primary	The number of spontaneous bleeding episodes per patient		Up to 36 months
Primary	The number of infusions of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes		Up to 36 months
Primary	The total dose of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes		Up to 36 months
Secondary	The number of infusions of Afstyla® required during the surgical procedures		Up to 36 months
Secondary	The total dose of Afstyla® required during the surgical procedures		Up to 36 months
Secondary	The incidence of adverse events (AE), severe AE, and AE related to Afstyla®		Up to 36 months