Clinical trial NCT04675541: Register of Patients With haEmophilia A tReated With Afstyla®
Official title: Observational Register of Patients With haEmophilia A tReated With Afstyla®
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Haemophilia A
Keywords: FVIII
Coagulation FVIII
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2020-12-19
Enrollment: 62
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Haemophilia A French patients 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.

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.

Primary outcomes:
measure: The annualized bleeding rate (spontaneous, traumatic) during long term prophylactic and on demand regimen.
timeFrame: Up to 36 months
measure: The number of spontaneous bleeding episodes per patient
timeFrame: Up to 36 months
measure: The number of infusions of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes
timeFrame: Up to 36 months
measure: The total dose of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes
timeFrame: Up to 36 months
Secondary outcomes:
measure: The number of infusions of Afstyla® required during the surgical procedures
timeFrame: Up to 36 months
measure: The total dose of Afstyla® required during the surgical procedures
timeFrame: Up to 36 months
measure: The incidence of adverse events (AE), severe AE, and AE related to Afstyla®
timeFrame: Up to 36 months
Other outcomes:
Locations:
city: Brest
country: France
facility: CHU Brest
city: Caen
country: France
facility: CHU Caen
city: Chambéry
country: France
facility: CHU Chambéry
city: Clermont-Ferrand
country: France
facility: CHU Clermont-Ferrand
city: Eaubonne
country: France
facility: Hôpital Simone Veil
city: Le Chesnay
country: France
facility: Hôpital Mignot
city: Le Kremlin-Bicêtre
country: France
facility: CRC-MHC Hôpital Kremlin Bicêtre
city: Lille
country: France
facility: CHRU Lille
city: Limoges
country: France
facility: CHU Limoges
city: Montpellier
country: France
facility: CHU Montpellier
city: Nantes
country: France
facility: CHU Nantes
city: Paris
country: France
facility: Hôpital Necker
city: Rennes
country: France
facility: CHU Rennes
city: Rouen
country: France
facility: CHU Rouen
city: Saint-Étienne
country: France
facility: CHU Saint-Etienne
city: Strasbourg
country: France
facility: Hôpital Hautepierre
city: Tours
country: France
facility: CHRU de Tours
Study officials:
affiliation: CSL Behring SA
name: Study Director
role: STUDY_DIRECTOR
References: