Haemophilia BB-SURENCT03655340
A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
An observational study of Haemophilia B, run by Swedish Orphan Biovitrum at 18 sites in France.
Trial summary
Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haemophilia B
- What is being tested?
- Alprolix
- How many people
- 91 participants
- Where
- 18 sitesFrance
- Study dates
- 12 Sep 2018to 16 Mar 2022
- 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: All eligible patients who present for a routine Clinical visit will be asked to participate in the study by the treating physician at participating haemophilia treatment centres in France.
Who may be able to join
- Have a diagnosis of haemophilia B and been treated previously with factor IX Product
- Have started Alprolix treatment prior to enrolment visit, or at enrolment prescribed treatment with Alprolix, regardless of participation in the study
- 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
- Participation in an investigational medicinal product trial at enrolment visit
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
France18 sites
Swedish Orphan Biovitrum Research Site (CHU de Bordeaux)
Bordeaux
Swedish Orphan Biovitrum Research Site
Brest
Swedish Orphan Biovitrum Research Site
Caen
Swedish Orphan Biovitrum Research Site
Chambéry
Swedish Orphan Biovitrum Research Site
Clermont-Ferrand
Swedish Orphan Biovitrum Research Site
Dijon
Swedish Orphan Biovitrum Research Site
La Réunion
Swedish Orphan Biovitrum Research Site
Lille
Swedish Orphan Biovitrum Research Site
Marseille
Swedish Orphan Biovitrum Research Site
Montpellier
Swedish Orphan Biovitrum Research Site
Nantes
Swedish Orphan Biovitrum Research Site
Nîmes
Swedish Orphan Biovitrum research site
Paris
Swedish Orphan Biovitrum research site
Poitiers
Swedish Orphan Biovitrum research site
Rennes
Swedish Orphan Biovitrum Research Site
Rouen
Swedish Orphan Biovitrum research site
Strasbourg
Swedish Orphan Biovitrum Research Site
Toulouse
About this study
Official title: A 24-month Prospective, Non-interventional, Multicentre Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B.
- Conditions
- Haemophilia B
- Being tested
- Alprolix
- Keywords
- Blood Coagulation Disorder, Hematologic Diseases, Coagulation Protein Disorder, Hemorrhagic Disorder, Genetic Diseases, Inborn
- Medical subject headings
- Hemophilia B, 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.
AlprolixDrug
Also called Eftrenonacog alfa, rFIXFc
Extended half-life factor IX product
| Study group | Role | What they receive |
|---|---|---|
| Prophylactic patients | Alprolix will be prescribed according to local practice and administered by patients with haemophilia B for prophylactic treatment Receives: Drug: Alprolix | |
| On demand patients | Alprolix will be prescribed according to local practice and administered by patients with haemophilia B for on-demand treatment Receives: Drug: Alprolix |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualised bleeding rate (ABR) (prophylactic treatment)
Assessed by diary
Measured: 24 months
Annualised injection frequency (prophylactic treatment)
Assessed by diary
Measured: 24 months
Annualised factor consumption (International Unit [IU]) (prophylactic treatment)
Assessed by diary
Measured: 24 months
Amount of factor Product used to treat a bleed (on-demand treatment)
Assessed by diary
Measured: 24 months
Number of injections to treat a bleed (on-demand treatment)
Assessed by diary
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 posted31 Aug 2018
- Study start12 Sep 2018
- Primary completion16 Mar 2022
- Study completion16 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Haemophilia BStudies 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
- 91 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Elena Santagostino, MD, Study director, Swedish Orphan Biovitrum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03655340
- Sponsor's protocol ID
- Sobi.Alprolix-001
- Acronym
- B-SURE
- Status last verified
- Sep 2024
- First submitted
- 30 Aug 2018
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Chambost H, Repesse Y, Genre-Volot F, Desprez D, Castet SM, Vanderbecken S, Zidi M, Gandossi C, Nuesch E, Palmborg H, Santagostino E; B-SURE Study Group. Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France. Ther Adv Hematol. 2025 Jan 26;16:20406207241311535. doi: 10.1177/20406207241311535. eCollection 2025.
PubMed 39872008
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 24-month Prospective, Non-interventional, Multicentre Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
- ClinicalTrials.gov ID
- NCT03655340
- Acronym / sponsor ID
- B-SURE / Sobi.Alprolix-001
- Conditions
- Haemophilia B
- Keywords
- Blood Coagulation Disorder, Hematologic Diseases, Coagulation Protein Disorder, Hemorrhagic Disorder, Genetic Diseases, Inborn
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 91 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- 12 Sep 2018 (actual)
- Primary completion
- 16 Mar 2022 (actual)
- Study completion
- 16 Mar 2022 (actual)
- First posted
- 31 Aug 2018 (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.
Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Prophylactic patients | – | Drug: AlprolixAlprolix will be prescribed according to local practice and administered by patients with haemophilia B for prophylactic treatment |
| On demand patients | – | Drug: AlprolixAlprolix will be prescribed according to local practice and administered by patients with haemophilia B for on-demand treatment |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All eligible patients who present for a routine Clinical visit will be asked to participate in the study by the treating physician at participating haemophilia treatment centres in France.
Key inclusion criteria (3 listed)
- Have a diagnosis of haemophilia B and been treated previously with factor IX Product
- Have started Alprolix treatment prior to enrolment visit, or at enrolment prescribed treatment with Alprolix, regardless of participation in the study
- 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 (1 listed)
- Participation in an investigational medicinal product trial at enrolment visit
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 |
|---|---|---|
| AlprolixAlso: Eftrenonacog alfa, rFIXFcExtended half-life factor IX product | Drug | On demand patients, Prophylactic patients |
Primary outcome
Annualised bleeding rate (ABR) (prophylactic treatment)
Time frame: 24 months
Assessed by diary
Annualised injection frequency (prophylactic treatment)
Time frame: 24 months
Assessed by diary
Annualised factor consumption (International Unit [IU]) (prophylactic treatment)
Time frame: 24 months
Assessed by diary
Amount of factor Product used to treat a bleed (on-demand treatment)
Time frame: 24 months
Assessed by diary
Number of injections to treat a bleed (on-demand treatment)
Time frame: 24 months
Assessed by diary
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 19 Sep 2024.
- Derived from this study: PubMed 39872008Chambost H, Repesse Y, Genre-Volot F, Desprez D, Castet SM, Vanderbecken S, Zidi M, Gandossi C, Nuesch E, Palmborg H, Santagostino E; B-SURE Study Group. Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France. Ther Adv Hematol. 2025 Jan 26;16:20406207241311535. doi: 10.1177/20406207241311535. eCollection 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.
Elena Santagostino, MD study director
Swedish Orphan Biovitrum
18 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 B, run by Swedish Orphan Biovitrum at 18 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 1 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 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 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?
Annualised bleeding rate (ABR) (prophylactic treatment) (time frame: 24 months). The study lists 5 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 NCT03655340, which also lists 1 linked publication.
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 19 Sep 2024.