Register of Patients With haEmophilia A tReated With Afstyla® (OPERA)
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|ClinicalTrials.gov Identifier: NCT04675541|
Recruitment Status : Active, not recruiting
First Posted : December 19, 2020
Last Update Posted : February 2, 2023
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|Condition or disease||Intervention/treatment|
|Haemophilia A||Biological: Afstyla®|
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|
|Actual Enrollment :||62 participants|
|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|
- Biological: Afstyla®
Solution for injectionOther Name: CSL627
- The annualized bleeding rate (spontaneous, traumatic) during long term prophylactic and on demand regimen. [ Time Frame: Up to 36 months ]
- The number of spontaneous bleeding episodes per patient [ Time Frame: Up to 36 months ]
- The number of infusions of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes [ Time Frame: Up to 36 months ]
- The total dose of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes [ Time Frame: Up to 36 months ]
- The number of infusions of Afstyla® required during the surgical procedures [ Time Frame: Up to 36 months ]
- The total dose of Afstyla® required during the surgical procedures [ Time Frame: Up to 36 months ]
- The incidence of adverse events (AE), severe AE, and AE related to Afstyla® [ Time Frame: Up to 36 months ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||Child, Adult, Older Adult|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
|Sampling Method:||Probability Sample|
- 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
- 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.
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
|Study Director:||Study Director||CSL Behring SA|
|Responsible Party:||CSL Behring|
|Other Study ID Numbers:||
2017-A03517-46 ( Other Identifier: ANSM )
|First Posted:||December 19, 2020 Key Record Dates|
|Last Update Posted:||February 2, 2023|
|Last Verified:||February 2023|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Coagulation Protein Disorders
Genetic Diseases, Inborn