Haemophilia AOPERANCT04675541
Register of Patients With haEmophilia A tReated With Afstyla®
An observational study of Haemophilia A, run by CSL Behring at 17 sites in France.
Trial 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 Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haemophilia A
- What is being tested?
- Afstyla®
- Type of treatment
- Injection or infusion
- How many people
- 62 participants
- Where
- 17 sitesFrance
- Study dates
- 24 Sep 2018to 31 Dec 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Haemophilia A French patients treated with Afstyla®
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
France17 sites
CHU Brest
Brest
CHU Caen
Caen
CHU Chambéry
Chambéry
CHU Clermont-Ferrand
Clermont-Ferrand
Hôpital Simone Veil
Eaubonne
Hôpital Mignot
Le Chesnay
CRC-MHC Hôpital Kremlin Bicêtre
Le Kremlin-Bicêtre
CHRU Lille
Lille
CHU Limoges
Limoges
CHU Montpellier
Montpellier
CHU Nantes
Nantes
Hôpital Necker
Paris
CHU Rennes
Rennes
CHU Rouen
Rouen
CHU Saint-Etienne
Saint-Etienne
Hôpital Hautepierre
Strasbourg
CHRU de Tours
Tours, 37044
About this study
Official title: 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
Read the 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.
- Conditions
- Haemophilia A
- Being tested
- Afstyla®
- Keywords
- FVIII, Coagulation FVIII
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Afstyla®Biological
Also called CSL627
Solution for injection
What is being measured
The primary outcome is the main question the study is designed to answer.
The annualized bleeding rate (spontaneous, traumatic) during long term prophylactic and on demand regimen.
Measured: Up to 36 months
The number of spontaneous bleeding episodes per patient
Measured: Up to 36 months
The number of infusions of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes
Measured: Up to 36 months
The total dose of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes
Measured: Up to 36 months
Secondary outcomes (3)
The number of infusions of Afstyla® required during the surgical procedures
Measured: Up to 36 months
The total dose of Afstyla® required during the surgical procedures
Measured: Up to 36 months
The incidence of adverse events (AE), severe AE, and AE related to Afstyla®
Measured: Up to 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2020
- Study start24 Sep 2018
- Primary completion31 Dec 2024
- Study completion31 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemophilia AStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 62 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, CSL Behring SA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04675541
- Sponsor's protocol ID
- OPERA study
- Other IDs
- 2017-A03517-46
- Acronym
- OPERA
- Status last verified
- Jan 2025
- First submitted
- 1 Dec 2020
- Last updated
- 8 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Register of Patients With haEmophilia A tReated With Afstyla®
- ClinicalTrials.gov ID
- NCT04675541
- Acronym / sponsor ID
- OPERA / OPERA study
- Conditions
- Haemophilia A
- Keywords
- FVIII, Coagulation FVIII
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 62 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- 24 Sep 2018 (actual)
- Primary completion
- 31 Dec 2024 (actual)
- Study completion
- 31 Dec 2024 (actual)
- First posted
- 19 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Haemophilia A French patients treated with Afstyla®
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Afstyla®Also: CSL627Solution for injection | Biological | – |
Primary outcome
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
3 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
CSL Behring SA
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Haemophilia A, run by CSL Behring at 17 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The annualized bleeding rate (spontaneous, traumatic) during long term prophylactic and on demand regimen. (time frame: Up to 36 months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2025.