Haemophilia APREVENTNCT03055611
A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B
An observational study of Haemophilia A and Haemophilia B, run by Swedish Orphan Biovitrum at 22 sites in Germany.
Trial summary
Elocta (rFVIIIFc) and Alprolix (rFIXFc) are recombinant extended half-life coagulation factor products. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Elocta and Alprolix in the prophylactic treatment of haemophilia A and B. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haemophilia A, Haemophilia B
- What is being tested?
- ELOCTA and ALPROLIX
- How many people
- 201 participants
- Where
- 22 sitesGermany
- Study dates
- 9 May 2017to 19 Apr 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 Elocta and Alprolix 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 Germany.
Who may be able to join
- Have a diagnosis of haemophilia A or B and previously treated with factor Product
- Have started prophylactic Elocta/Alprolix treatment prior to enrollment visit, or at enrollment prescribed prophylactic treatment with Elocta or 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, from four weeks prior to first injection with Elocta or Alprolix to enrollment visit.
Trial locations and contacts
22 sites in 1 country.
22 sites in 1 country
Germany22 sites
Swedish Orphan Biovitrum Reserach site
Berlin
Swedish Orphan Biovitrum Reserach Site
Blaubeuren Abbey
Swedish Orphan Biovitrum Research Site
Bonn
Swedish Orphan Biovitrum Research Site
Delmenhorst
Swedish Orphan Biovitrum Research Site
Duisburg
Swedish Orphan Biovitrum Reserach site
Erlangen
Swedish Orphan Biovitrum Research Site
Frankfurt
Swedish Orphan Biovitrum Reserach site
Frankfurt
Swedish Orphan Biovitrum Research Site
Fürth
Swedish Orphan Biovitrum Research Site
Hamburg
Swedish Orphan Biovitrum Research Site
Hanover
Swedish Orphan Biovitrum Reserach site
Hanover
Swedish Orphan Biovitrum Reserach site
Heidelberg
Swedish Orphan Biovitrum Reserach site
Homburg
Swedish Orphan Biovitrum Research Site
Jena
Swedish Orphan Biovitrum Reserach Site
Jena
Swedish Orphan Biovitrum Reserach site
Leipzig
Swedish Orphan Biovitrum Reserach Site
Mörfelden-Walldorf
Swedish Orphan Biovitrum Research Site
München
Swedish Orphan Biovitrum Reserach site
München
Swedish Orphan Biovitrum Reserach site
Münster
Swedish Orphan Biovitrum Research Site
Würzburg
About this study
Official title: A 24-month Prospective, Non-interventional, Multicentre Study to Evaluate the Real-World Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B
Elocta (rFVIIIFc) and Alprolix (rFIXFc) are recombinant extended half-life coagulation factor products. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Elocta and Alprolix in the prophylactic treatment of haemophilia A and B.
- Conditions
- Haemophilia A, Haemophilia B
- Being tested
- ELOCTA, ALPROLIX
- Keywords
- Blood Coagulation Disorder, Hematologic Diseases, Coagulation Protein Disorder, Hemorrhagic Disorder, Genetic Diseases, Inborn
- Medical subject headings
- Hemophilia A, 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.
ELOCTADrug
Also called Eloctate, efmoroctocog alfa, rFVIIIFc
Extended half-life factor VIII product
ALPROLIXDrug
Also called Eftrenonacog alfa, rFIXFc
Extended half-life factor IX product
| Study group | Role | What they receive |
|---|---|---|
| Haemophilia A patients | Elocta will be prescribed according to local practice and administered by patients with haemophilia A for prophylactic treatment Receives: Drug: ELOCTA | |
| Haemophilia B patients | Alprolix will be prescribed according to local practice and administered by patients with haemophilia B for prophylactic 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)
Based on bleeding episodes assessed by local practice
Measured: 24 months
Annualised injection frequency
Assessed by prescription
Measured: 24 months
Annualised factor consumption (IU)
Assessed by dispensed factor product
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 posted16 Feb 2017
- Study start9 May 2017
- Primary completion19 Apr 2022
- Study completion19 Apr 2022
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.
- 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
- 201 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Physician, MD, Study director, Swedish Orphan Biovitrum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03055611
- Sponsor's protocol ID
- Sobi.HAEM89-002
- Acronym
- PREVENT
- Status last verified
- Oct 2024
- First submitted
- 14 Feb 2017
- Last updated
- 2 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
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
- A 24-month Prospective, Non-interventional, Multicentre Study to Evaluate the Real-World Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B
- ClinicalTrials.gov ID
- NCT03055611
- Acronym / sponsor ID
- PREVENT / Sobi.HAEM89-002
- Conditions
- Haemophilia A, Haemophilia B
- Keywords
- Blood Coagulation Disorder, Hematologic Diseases, Coagulation Protein Disorder, Hemorrhagic Disorder, Genetic Diseases, Inborn
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 201 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- 9 May 2017 (actual)
- Primary completion
- 19 Apr 2022 (actual)
- Study completion
- 19 Apr 2022 (actual)
- First posted
- 16 Feb 2017 (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.
Elocta (rFVIIIFc) and Alprolix (rFIXFc) are recombinant extended half-life coagulation factor products. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Elocta and Alprolix in the prophylactic treatment of haemophilia A and B.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Haemophilia A patients | – | Drug: ELOCTAElocta will be prescribed according to local practice and administered by patients with haemophilia A for prophylactic treatment |
| Haemophilia B patients | – | Drug: ALPROLIXAlprolix will be prescribed according to local practice and administered by patients with haemophilia B for prophylactic treatment |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All eligible Elocta and Alprolix 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 Germany.
Key inclusion criteria (3 listed)
- Have a diagnosis of haemophilia A or B and previously treated with factor Product
- Have started prophylactic Elocta/Alprolix treatment prior to enrollment visit, or at enrollment prescribed prophylactic treatment with Elocta or 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, from four weeks prior to first injection with Elocta or Alprolix to enrollment 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 |
|---|---|---|
| ELOCTAAlso: Eloctate, efmoroctocog alfa, rFVIIIFcExtended half-life factor VIII product | Drug | Haemophilia A patients |
| ALPROLIXAlso: Eftrenonacog alfa, rFIXFcExtended half-life factor IX product | Drug | Haemophilia B patients |
Primary outcome
Annualised bleeding rate (ABR)
Time frame: 24 months
Based on bleeding episodes assessed by local practice
Annualised injection frequency
Time frame: 24 months
Assessed by prescription
Annualised factor consumption (IU)
Time frame: 24 months
Assessed by dispensed factor product
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.
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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
Swedish Orphan Biovitrum
22 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 and Haemophilia B, run by Swedish Orphan Biovitrum at 22 sites in Germany.
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 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 22 sites in Germany. 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?
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; AzTrials last refreshed its copy on 28 Oct 2026.