Hemophilia ANCT02976753
Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products
An observational study of Hemophilia A, run by Swedish Orphan Biovitrum at 36 sites in 9 countries.
Trial summary
The purpose of the study is to evaluate the effectiveness of Elocta compared to conventional factor products in the prophylactic treatment of patients with haemophilia A over a 24-month prospective period. Data will also be collected for a 12 month retrospective period. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- efmoroctocog alfa and Factor VIII
- How many people
- 361 participants
- Where
- 36 sitesin 9 countries
- Study dates
- Dec 2016to 3 Nov 2021
- 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
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Male patients with a diagnosis of haemophilia A prescribed prophylactic treatment with Elocta or conventional factor product
Who may be able to join
- Male patients with a diagnosis of haemophilia A
- Received prophylactic treatment with a factor product for management of haemophilia A in the 12 months prior to enrolment
- At enrolment prescribed prophylactic treatment with Elocta or conventional factor product regardless of participation in the study
- Having at least 12 months documented pre-study treatment data regarding prophylactic treatment prescriptions and bleeding episodes prior to the baseline visit
- Signed and dated informed consent provided by the patient or the patient's legally acceptable representative for patients under the legal age before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations
Who cannot join
- Enrolment in a concurrent clinical interventional study involving intake of an investigational medicinal product (IMP), within 1 year prior to enrolment
- Previously treated with commercially available extended half-life products other than Elocta
- Presence of factor VIII inhibitors (≥0.60 Bethesda Unit [BU]/mL ) at the latest available inhibitor test using the Nijmegen modified Bethesda assay
Trial locations and contacts
36 sites in 9 countries.
36 sites in 9 countries
Italy9 sites
Swedish Orphan Biovitrum Reserach site
Catanzaro
Swedish Orphan Biovitrum Reserach site
Florence
Swedish Orphan Biovitrum Research site
Milan
Swedish Orphan Biovitrum Reserach site
Naples
Swedish Orphan Biovitrum Research Site
Padova
Swedish Orphan Biovitrum Reserach site
Palermo
Swedish Orphan Biovitrum Reserach site
Parma
Swedish Orphan Biovitrum Reserach site
Rome
Swedish Orphan Biovitrum Research site
Turin
United Kingdom7 sites
Swedish Orphan Biovitrum Research site
Birmingham
Swedish Orphan Biovitrum Research site
Canterbury
Swedish Orphan Biovitrum Research site
Liverpool
Swedish Orphan Biovitrum Reserach site
London
Swedish Orphan Biovitrum Research site
Manchester
Swedish Orphan Biovitrum Research site
Sheffield
Swedish Orphan Biovitrum Research site
Southampton
Spain6 sites
Swedish Orphan Biovitrum research site
Alicante
Swedish Orphan Biovitrum Reserach site
Barcelona
Swedish Orphan Biovitrum Reserach site
Madrid
Swedish Orphan Biovitrum Research site
Málaga
Swedish Orphan Biovitrum Reserach site
Seville
Swedish Orphan Biovitrum Research site
Valencia
Austria4 sites
Swedish Orphan Biovitrum Research site
Innsbruck
Swedish Orphan Biovitrum Research site
Linz
Swedish Orphan Biovitrum Research Site
Sankt Pölten
Swedish Orphan Biovitrum Research site
Vienna
France4 sites
Swedish Orphan Biovitrum Research site
Caen
Swedish Orphan Biovitrum Research site
Montpellier
Swedish Orphan Biovitrum Research site
Nancy
Swedish Orphan Biovitrum Research site
Rennes
Sweden3 sites
Swedish Orphan Biovitrum Research site
Gothenburg
Swedish Orphan Biovitrum Research site
Malmö
Swedish Orphan Biovitrum Research site
Stockholm
About this study
Official title: 24-month Prospective, Multicentre, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products in the Prophylactic Treatment of Patients With Haemophilia A (A-SURE)
The purpose of the study is to evaluate the effectiveness of Elocta compared to conventional factor products in the prophylactic treatment of patients with haemophilia A over a 24-month prospective period. Data will also be collected for a 12 month retrospective period.
- Conditions
- Hemophilia A
- Being tested
- efmoroctocog alfa, Factor VIII
- Keywords
- Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn
- Medical subject headings
- Hemophilia A, Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
efmoroctocog alfaDrug
Also called Elocta
extended half-life factor VIII product
Factor VIIIDrug
conventional factor VIII product
| Study group | Role | What they receive |
|---|---|---|
| Elocta | Elocta will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice Receives: Drug: efmoroctocog alfa | |
| Conventional factor VIII product | Conventional factor VIII products will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice Receives: Drug: Factor VIII |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualised bleeding rate (ABR)
Measured: 24 months
Annualised injection frequency
Measured: 24 months
Annualised factor consumption (International Unit [IU])
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2016 (estimated)
- Study startDec 2016
- Primary completion3 Nov 2021
- Study completion3 Nov 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- 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
- 361 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Responsible party
- Sponsor
- Investigators
- Study Physician, MD, Study director, Sweden Orphan Biovitrum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02976753
- Sponsor's protocol ID
- Sobi.Elocta-002
- Status last verified
- Oct 2024
- First submitted
- 24 Nov 2016
- Last updated
- 2 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Publications
Oldenburg J, Hay C, Peyvandi F, Lehtinen AE, Pabinger I, Nuesch E, Malmstrom H, Bednar E, Lethagen S. A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A. Hematology. 2025 Dec;30(1):2513186. doi: 10.1080/16078454.2025.2513186. Epub 2025 Jul 4.
PubMed 40616274Oldenburg J, Hay CRM, Jimenez-Yuste V, Peyvandi F, Schved JF, Szamosi J, Winding B, Lethagen S. Design of a prospective observational study on the effectiveness and real-world usage of recombinant factor VIII Fc (rFVIIIFc) compared with conventional products in haemophilia A: the A-SURE study. BMJ Open. 2019 May 30;9(5):e028012. doi: 10.1136/bmjopen-2018-028012.
PubMed 31152037
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 24-month Prospective, Multicentre, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products in the Prophylactic Treatment of Patients With Haemophilia A (A-SURE)
- ClinicalTrials.gov ID
- NCT02976753
- Acronym / sponsor ID
- Sobi.Elocta-002
- Conditions
- Hemophilia A
- Keywords
- Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 361 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Study start
- Dec 2016 (actual)
- Primary completion
- 3 Nov 2021 (actual)
- Study completion
- 3 Nov 2021 (actual)
- First posted
- 29 Nov 2016 (estimated)
- 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.
The purpose of the study is to evaluate the effectiveness of Elocta compared to conventional factor products in the prophylactic treatment of patients with haemophilia A over a 24-month prospective period. Data will also be collected for a 12 month retrospective period.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Elocta | – | Drug: efmoroctocog alfaElocta will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice |
| Conventional factor VIII product | – | Drug: Factor VIIIConventional factor VIII products will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Male patients with a diagnosis of haemophilia A prescribed prophylactic treatment with Elocta or conventional factor product
Key inclusion criteria (5 listed)
- Male patients with a diagnosis of haemophilia A
- Received prophylactic treatment with a factor product for management of haemophilia A in the 12 months prior to enrolment
- At enrolment prescribed prophylactic treatment with Elocta or conventional factor product regardless of participation in the study
- Having at least 12 months documented pre-study treatment data regarding prophylactic treatment prescriptions and bleeding episodes prior to the baseline visit
- Signed and dated informed consent provided by the patient or the patient's legally acceptable representative for patients under the legal age before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations
Key exclusion criteria (3 listed)
- Enrolment in a concurrent clinical interventional study involving intake of an investigational medicinal product (IMP), within 1 year prior to enrolment
- Previously treated with commercially available extended half-life products other than Elocta
- Presence of factor VIII inhibitors (≥0.60 Bethesda Unit [BU]/mL ) at the latest available inhibitor test using the Nijmegen modified Bethesda assay
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 |
|---|---|---|
| efmoroctocog alfaAlso: Eloctaextended half-life factor VIII product | Drug | Elocta |
| Factor VIIIconventional factor VIII product | Drug | Conventional factor VIII product |
Primary outcome
Annualised bleeding rate (ABR)
Time frame: 24 months
Annualised injection frequency
Time frame: 24 months
Annualised factor consumption (International Unit [IU])
Time frame: 24 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 2 Oct 2024.
- Derived from this study: PubMed 40616274Oldenburg J, Hay C, Peyvandi F, Lehtinen AE, Pabinger I, Nuesch E, Malmstrom H, Bednar E, Lethagen S. A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A. Hematology. 2025 Dec;30(1):2513186. doi: 10.1080/16078454.2025.2513186. Epub 2025 Jul 4.
- Derived from this study: PubMed 31152037Oldenburg J, Hay CRM, Jimenez-Yuste V, Peyvandi F, Schved JF, Szamosi J, Winding B, Lethagen S. Design of a prospective observational study on the effectiveness and real-world usage of recombinant factor VIII Fc (rFVIIIFc) compared with conventional products in haemophilia A: the A-SURE study. BMJ Open. 2019 May 30;9(5):e028012. doi: 10.1136/bmjopen-2018-028012.
- Individual participant dataSharing planned: Undecided
Related trials for Hemophilia A
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A Research Study Investigating Denecimig (Mim8) in Adults With Acquired Haemophilia A
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A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
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An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
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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 Physician, MD study director
Sweden Orphan Biovitrum
36 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Swedish Orphan Biovitrum at 36 sites in 9 countries.
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 5 inclusion and 3 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 2 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 36 sites in Italy, United Kingdom, Spain, Austria, France and 4 more countries. 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?
Annualised bleeding rate (ABR) (time frame: 24 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02976753, which also lists 2 linked publications.
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 2 Oct 2024.