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

Observational Register of Patients With haEmophilia A tReated With Afstyla®

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

Study Overview

• Status: Active, not recruiting
• Conditions: Haemophilia A
• Intervention / Treatment: Biological: Afstyla®

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
• Enrollment (Actual): 62

Contacts and Locations

Study Locations

• France
  • Brest, France
    • CHU Brest
  • Caen, France
    • CHU caen
  • Chambéry, France
    • CHU Chambéry
  • Clermont-Ferrand, France
    • CHU Clermont-Ferrand
  • Eaubonne, France
    • Hôpital Simone Veil
  • Le Chesnay, France
    • Hôpital Mignot
  • Le Kremlin-Bicêtre, France
    • CRC-MHC Hôpital Kremlin Bicêtre
  • Lille, France
    • CHRU Lille
  • Limoges, France
    • Chu Limoges
  • Montpellier, France
    • CHU Montpellier
  • Nantes, France
    • CHU Nantes
  • Paris, France
    • Hôpital Necker
  • Rennes, France
    • CHU Rennes
  • Rouen, France
    • CHU Rouen
  • Saint-Étienne, France
    • CHU Saint-Etienne
  • Strasbourg, France
    • Hôpital Hautepierre
  • Tours, France, 37044
    • CHRU de Tours

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Haemophilia A French patients treated with Afstyla®

Description

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.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The annualized bleeding rate (spontaneous, traumatic) during long term prophylactic and on demand regimen.	Up to 36 months
The number of spontaneous bleeding episodes per patient	Up to 36 months
The number of infusions of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes	Up to 36 months
The total dose of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes	Up to 36 months

Secondary Outcome Measures

Outcome Measure	Time Frame
The number of infusions of Afstyla® required during the surgical procedures	Up to 36 months
The total dose of Afstyla® required during the surgical procedures	Up to 36 months
The incidence of adverse events (AE), severe AE, and AE related to Afstyla®	Up to 36 months

Collaborators and Investigators

• Sponsor: CSL Behring
• Investigators: Study Director: Study Director, CSL Behring SA

Study record dates

Study Major Dates

• Study Start (Actual): September 24, 2018
• Primary Completion (Estimated): December 1, 2024
• Study Completion (Estimated): December 1, 2024

Study Registration Dates

• First Submitted: December 1, 2020
• First Submitted That Met QC Criteria: December 15, 2020
• First Posted (Actual): December 19, 2020

Study Record Updates

• Last Update Posted (Actual): April 19, 2024
• Last Update Submitted That Met QC Criteria: April 18, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: FVIII; Coagulation FVIII
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: OPERA study; 2017-A03517-46 (Other Identifier: ANSM)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No