Register of Patients With haEmophilia A tReated With Afstyla®

NCT ID: NCT04675541

Last Updated: 2025-01-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

62 participants

Study Classification

OBSERVATIONAL

Study Start Date

2018-09-24

Study Completion Date

2024-12-31

Brief Summary

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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

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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.

Conditions

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Haemophilia A

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Interventions

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Afstyla®

Solution for injection

Intervention Type BIOLOGICAL

Other Intervention Names

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CSL627

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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CSL Behring

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

CSL Behring SA

Locations

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CHU Brest

Brest, , France

Site Status

CHU Caen

Caen, , France

Site Status

CHU Chambéry

Chambéry, , France

Site Status

CHU Clermont-Ferrand

Clermont-Ferrand, , France

Site Status

Hôpital Simone Veil

Eaubonne, , France

Site Status

Hôpital Mignot

Le Chesnay, , France

Site Status

CRC-MHC Hôpital Kremlin Bicêtre

Le Kremlin-Bicêtre, , France

Site Status

CHRU Lille

Lille, , France

Site Status

CHU Limoges

Limoges, , France

Site Status

CHU Montpellier

Montpellier, , France

Site Status

CHU Nantes

Nantes, , France

Site Status

Hôpital Necker

Paris, , France

Site Status

CHU Rennes

Rennes, , France

Site Status

CHU Rouen

Rouen, , France

Site Status

CHU Saint-Etienne

Saint-Etienne, , France

Site Status

Hôpital Hautepierre

Strasbourg, , France

Site Status

CHRU de Tours

Tours, , France

Site Status

Countries

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France

Other Identifiers

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2017-A03517-46

Identifier Type: OTHER

Identifier Source: secondary_id

OPERA study

Identifier Type: -

Identifier Source: org_study_id

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