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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04675541
Recruitment Status : Active, not recruiting
First Posted : December 19, 2020
Last Update Posted : February 2, 2023
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Study Description
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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

Condition or disease Intervention/treatment
Haemophilia A 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 Design
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Study Type : Observational
Actual Enrollment : 62 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Observational Register of Patients With haEmophilia A tReated With Afstyla®
Actual Study Start Date : September 24, 2018
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Groups and Cohorts
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Intervention Details:
  • Biological: Afstyla®
    Solution for injection
    Other Name: CSL627

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

Secondary Outcome Measures :
  1. The number of infusions of Afstyla® required during the surgical procedures [ Time Frame: Up to 36 months ]
  2. The total dose of Afstyla® required during the surgical procedures [ Time Frame: Up to 36 months ]
  3. The incidence of adverse events (AE), severe AE, and AE related to Afstyla® [ Time Frame: Up to 36 months ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Haemophilia A French patients treated with Afstyla®
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04675541


Locations
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Sponsors and Collaborators
CSL Behring
Investigators
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Study Director: Study Director CSL Behring SA
More Information
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
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Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT04675541    
Other Study ID Numbers: OPERA study
2017-A03517-46 ( Other Identifier: ANSM )
First Posted: December 19, 2020    Key Record Dates
Last Update Posted: February 2, 2023
Last Verified: February 2023

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by CSL Behring:
FVIII
Coagulation FVIII
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn