Hemophilia AA-MORENCT04293523
A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
An observational study of Hemophilia A, run by Swedish Orphan Biovitrum at 53 sites in 14 countries.
Trial summary
This is a 48-month observational, prospective, multicentre study. The overall aim of the study is to evaluate the long-term effectiveness of Elocta treatment on joint health in patients treated prophylactically with Elocta in a real-world setting. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A
- What is being tested?
- ELOCTA
- How many people
- 427 participants
- Where
- 53 sitesin 14 countries
- Study dates
- 30 Mar 2020to 4 May 2026
- 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: Patients diagnosed with haemophilia A regardless of severity, of any gender and age, on factor treatment with Elocta according to usual clinical practice.
Who may be able to join
- Provided signed and dated informed consent by the patient, or the patient's legally authorized representative(s) for patients under the legal age, before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations
- Have a diagnosis of haemophilia A
- At enrolment on prophylactic treatment with Elocta, independent of participation in the study
Who cannot join
- Enrolment in another concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to inclusion in this study
- Presence of factor VIII antibodies (inhibitors) (≥0.60 Bethesda Unit [BU]/mL) at the latest available inhibitor test
Trial locations and contacts
53 sites in 14 countries.
53 sites in 14 countries
Spain12 sites
Hospital de la Santa Creu i Sant Pau
Barcelona
Hospital Vall d'Hebron
Barcelona
Sant Johan De Deu
Barcelona
Hospital Universitario Cruces
Cruces
Hospital Universitario Donostia
Donostia / San Sebastian
Hospital Universitario Carlos Haya
Málaga
Hospital Virgen de la Arrixaca
Murcia
Complejo Hospitalario de Navarra
Navarro
Hospital Universitario Central de Asturias (HUCA)
Oviedo
Hospital Universitario Son Espases
Palma de Mallorca
Hospital Universitario Virgen del Rocío
Seville
Hospital Alvaro Cunqueiro
Vigo
Germany10 sites
Charité-Universitätsmedizin Berlin Campus Virchow Klinikum
Berlin
Universitätsklinikum Bonn AöR, Institut für Experimentelle Hämatologie und Transfusionsmedizin
Bonn
Hämostaseologie/Hämophiliezentrum, Medizinische Klinik 2 Institut für Transfusionsmedizin, Universitätsklinikum
Frankfurt
Universitätsklinikum Frankfurt - Klinik für Kinder- und Jugendmedizin
Frankfurt
Universitätsklinikum Hamburg-Eppendorf (UKE)
Hamburg
Medizinische Hochschule Hannover
Hanover
Werlhof-Institut für Hämostaseologie GmbH
Hanover
SRH-Klinikum Heidelberg
Heidelberg
HZRM Hämöphilie Zentrum Rhein Main
Mörfelden-Walldorf
Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital am Universitätsklinikum München
München
Italy7 sites
University of Bari Aldo Moro (Centro Emofilia Policlinico - Pediatria U.O.)
Bari
AUSL Romagna Centro Emofilia U.O.C., Medicina Trasfusionale Dipartimento Patologia, Clinica Ospedale M. Bufalini
Cesena
Giannina Gaslini Institute
Genova
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan
University Hospital of Parma, AOUP, Haemophilia Center
Parma
Uo Malattie Emorragiche e Trombotiche Fondazione Policlinico Universitario Agostino Gemelli, IRCCS, Universitá Catolica del Sacro Coure
Rome
Azienda Ospedaliera Città della Salute e della Scienza di Torino Regina Margherita
Torino
Saudi Arabia4 sites
Dr Suliman Al Habib Hospital Riyadh
Riyadh
King Faisal Specialised Hospital, KFSH Riyadh, Children
Riyadh
King Faisal Specialist Hospital KFSH, Adults
Riyadh
Riyadh Military Hospital (P.S.M.C)
Riyadh
Switzerland4 sites
Universitätsklinik für Hämatologie und Hämatologisches Zentrallabor Inselspital
Bern
Service et Laboratoire central d'hématologie, Adults
Lausanne
Zentrum für Labormedizin
Sankt Gallen
Universitätsspital Zürich Klinik für Medizinische Onkologie und Hämatologie
Zurich
Czechia3 sites
University Hospital Brno
Brno
University Hospital Ostrava
Ostrava
Dept. of Pediatric Haematology and Oncology, University Hospital Motol
Prague
Estonia2 sites
Lastehaigla, Tallinn (Tallinn Children´s Hospital)
Tallinn
The North Estonia Medical Centre Hematoloogiakeskus, Regionalhaigla
Tallinn
Finland2 sites
Helsinki University Central Hospital, New Children Hospital
Helsinki
Turku University Central Hospital, Paediatric and adolescent haematology and oncology clinic
Turku
Greece2 sites
Ippokrateio Hospital Thessaloniki (adult department)
Thessaloniki
Ippokrateio Hospital Thessaloniki (pediatric department)
Thessaloniki
Sweden2 sites
Hematologimottagning Sahlgrenska
Gothenburg
Department of Haematology, Oncology and Radiation Physics, Skåne University Hospital Malmö
Malmö
United Kingdom2 sites
East Kent Hospitals University NHS Foundation Trust, Kent Haemophilia and Thrombosis Centre, Kent and Canterbury Hospital
Canterbury
Great Ormond Street Hospital, Royal London Hospital for Integrated Medicine
London
Slovenia1 site
University Medical Centre Ljubljana Division of Paediatrics
Ljubljana
About this study
Official title: A 48-Month, Multi-Centre, Observational Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
This is a 48-month observational, prospective, multicentre study. The overall aim of the study is to evaluate the long-term effectiveness of Elocta treatment on joint health in patients treated prophylactically with Elocta in a real-world setting.
Read the detailed description
Haemophilia A is a rare genetic disorder estimated to occur in one out of 10,000 live births, characterized by a deficiency in coagulation factor VIII causing impaired haemostasis and prolonged bleeding episodes. Moderate haemophilia, defined as < 5%, and severe haemophilia, defined as < 1% of normal factor VIII activity result in frequent and spontaneous bleeds into muscles and joints, commonly the elbows, knees, and ankles. Bleeding into joints can cause acute pain and swelling and can result in reduced joint range of motion, long-term cartilage damage and debilitating haemophilic arthropathy. Early use of prophylaxis with factor VIII replacement is recommended following diagnosis of haemophilia A to maintain joint health and prevent joint destruction. However, despite the use of prophylaxis many patients still experience joint bleeds which may lead to joint deterioration over time. The risk of joint bleeds increases with the amount of time spent below certain FVIII trough levels, e.g. 1, 3 or 5 IU/dL. Thus, there is probably a relation between the intensity of the prophylactic treatment regimen and joint health. Elocta is an extended half-life rFVIII product (EHL rFVIII), with a slower clearance as compared to conventional FVIII products. Treatment with Elocta will therefore provide the treater with a greater flexibility for individualizing prophylaxis as compared to conventional FVIII. Higher trough levels can be reached with Elocta without increasing factor usage or injection frequency. The treater can instead choose to reduce the injection frequency or the factor consumption without lowering trough levels.
Patients may limit their physical activities due to fear of bleeding if they are unaware of their current FVIII level. Patient apps and wearables are now available which allow patients to view their predicted FVIII levels, and capture health-related data (such as bleedings, pain, well-being, physical activity levels etc.). This data can be shared with the treating physician supporting the planning to individualize the patient's factor treatment based on current lifestyle, health status and physical activity levels. Florio, a certified medical device used as part of routine clinical practice, is such an app, and the data output and patient feedback on their activity levels and sense of protection while using Florio will be analysed as exploratory objectives in this study.
The main purpose of this study is to evaluate the effectiveness of Elocta on joint health over a long observation period (48 months). The study will also explore the influence on long term joint health of different Elocta prophylaxis regimens leading to different trough levels and if the extent of patients' physical activity levels can be associated with predicted FVIII levels.
- Conditions
- Hemophilia A
- Being tested
- ELOCTA
- Keywords
- Joint health, HEAD-US, HJHS
- Medical subject headings
- Hemophilia A
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
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A patients | All patients diagnosed with haemophilia A regardless of severity, on factor treatment with Elocta according to usual clinical practice. Receives: Drug: ELOCTA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Joint health: Target joint development
Number of target joints
Measured: 48 months
Joint health: Target joint resolution
Number of resolved target joints
Measured: 48 months
Joint health: Target joint recurrence
Number of recurring target joints
Measured: 48 months
Joint health: Annualised joint bleeding rate (AJBR) for treated bleeds
Number of joint bleeding events per year, for treated bleeds
Measured: 48 months
Secondary outcomes (3)
Disease Activity (hypertrophic synovium) and Disease Damage (Cartilage or Bone) scores for elbows, knees and ankles
The Haemophilia Early Arthropathy Detection with UltraSound (HEAD-US) protocol will be used. The total score represents the sum of item scores for abnormalities detected. Its values range from 0 (minimum) to 8 (maximum). A higher score indicates a worse outcome.
Measured: 48 months
Global Gait Score, and/or joint score items for elbows, knees and ankles.
The Haemophilia Joint Health Score (HJHS) system will be used. Global Gait Score, and/or Joint score items (swelling, duration of swelling, muscle atrophy, axial alignment, crepitus on motion, flexion loss, instability, extension loss, joint pain, strength, gait) for elbows, knees and ankles.
The minimum score per joint is 0, the maximum score is 20. The overall total joint score (range 0-120) is the sum of the 6 six index joint (elbows, knees and ankles) scores. A higher score indicates a worse outcome.
Measured: 48 months
Joint and physical evaluation for elbows, knees and ankles.
The WFH Physical Examination Score (AKA Gilbert Score) will be used. Joint evaluation (pain, bleeding, physical examination and radiologic evaluation) and physical evaluation (swelling, muscle atrophy, axial deformity, crepitus on motion, range of motion, flexion contracture, and instability). A higher score indicates a worse outcome.
Measured: 48 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2020
- Study start30 Mar 2020
- Primary completion4 May 2026
- Study completion4 May 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 427 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Physician, Study director, Swedish Orphan Biovitrum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04293523
- Sponsor's protocol ID
- Sobi.Elocta-005
- Acronym
- A-MORE
- Status last verified
- Sep 2026
- First submitted
- 20 Feb 2020
- Last updated
- 22 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 48-Month, Multi-Centre, Observational Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
- ClinicalTrials.gov ID
- NCT04293523
- Acronym / sponsor ID
- A-MORE / Sobi.Elocta-005
- Conditions
- Hemophilia A
- Keywords
- Joint health, HEAD-US, HJHS
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 427 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- 30 Mar 2020 (actual)
- Primary completion
- 4 May 2026 (actual)
- Study completion
- 4 May 2026 (actual)
- First posted
- 3 Mar 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.
This is a 48-month observational, prospective, multicentre study. The overall aim of the study is to evaluate the long-term effectiveness of Elocta treatment on joint health in patients treated prophylactically with Elocta in a real-world setting.
Detailed description
Haemophilia A is a rare genetic disorder estimated to occur in one out of 10,000 live births, characterized by a deficiency in coagulation factor VIII causing impaired haemostasis and prolonged bleeding episodes. Moderate haemophilia, defined as < 5%, and severe haemophilia, defined as < 1% of normal factor VIII activity result in frequent and spontaneous bleeds into muscles and joints, commonly the elbows, knees, and ankles. Bleeding into joints can cause acute pain and swelling and can result in reduced joint range of motion, long-term cartilage damage and debilitating haemophilic arthropathy. Early use of prophylaxis with factor VIII replacement is recommended following diagnosis of haemophilia A to maintain joint health and prevent joint destruction. However, despite the use of prophylaxis many patients still experience joint bleeds which may lead to joint deterioration over time. The risk of joint bleeds increases with the amount of time spent below certain FVIII trough levels, e.g. 1, 3 or 5 IU/dL. Thus, there is probably a relation between the intensity of the prophylactic treatment regimen and joint health. Elocta is an extended half-life rFVIII product (EHL rFVIII), with a slower clearance as compared to conventional FVIII products. Treatment with Elocta will therefore provide the treater with a greater flexibility for individualizing prophylaxis as compared to conventional FVIII. Higher trough levels can be reached with Elocta without increasing factor usage or injection frequency. The treater can instead choose to reduce the injection frequency or the factor consumption without lowering trough levels.
Patients may limit their physical activities due to fear of bleeding if they are unaware of their current FVIII level. Patient apps and wearables are now available which allow patients to view their predicted FVIII levels, and capture health-related data (such as bleedings, pain, well-being, physical activity levels etc.). This data can be shared with the treating physician supporting the planning to individualize the patient's factor treatment based on current lifestyle, health status and physical activity levels. Florio, a certified medical device used as part of routine clinical practice, is such an app, and the data output and patient feedback on their activity levels and sense of protection while using Florio will be analysed as exploratory objectives in this study.
The main purpose of this study is to evaluate the effectiveness of Elocta on joint health over a long observation period (48 months). The study will also explore the influence on long term joint health of different Elocta prophylaxis regimens leading to different trough levels and if the extent of patients' physical activity levels can be associated with predicted FVIII levels.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A patients | – | Drug: ELOCTAAll patients diagnosed with haemophilia A regardless of severity, on factor treatment with Elocta according to usual clinical practice. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients diagnosed with haemophilia A regardless of severity, of any gender and age, on factor treatment with Elocta according to usual clinical practice.
Key inclusion criteria (3 listed)
- Provided signed and dated informed consent by the patient, or the patient's legally authorized representative(s) for patients under the legal age, before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations
- Have a diagnosis of haemophilia A
- At enrolment on prophylactic treatment with Elocta, independent of participation in the study
Key exclusion criteria (2 listed)
- Enrolment in another concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to inclusion in this study
- Presence of factor VIII antibodies (inhibitors) (≥0.60 Bethesda Unit [BU]/mL) at the latest available inhibitor test
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 | Hemophilia A patients |
Primary outcome
Joint health: Target joint development
Time frame: 48 months
Number of target joints
Joint health: Target joint resolution
Time frame: 48 months
Number of resolved target joints
Joint health: Target joint recurrence
Time frame: 48 months
Number of recurring target joints
Joint health: Annualised joint bleeding rate (AJBR) for treated bleeds
Time frame: 48 months
Number of joint bleeding events per year, for treated bleeds
3 secondary outcomes
Disease Activity (hypertrophic synovium) and Disease Damage (Cartilage or Bone) scores for elbows, knees and ankles
Time frame: 48 months
Global Gait Score, and/or joint score items for elbows, knees and ankles.
Time frame: 48 months
Joint and physical evaluation for elbows, knees and ankles.
Time frame: 48 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 22 Sep 2026.
- Individual participant dataSharing planned: No
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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 study director
Swedish Orphan Biovitrum
53 sites in 14 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 53 sites in 14 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 3 inclusion and 2 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 22 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 53 sites in Spain, Germany, Italy, Saudi Arabia, Switzerland and 9 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?
Joint health: Target joint development (time frame: 48 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 22 Sep 2026.