An observational study in Hemophilia A, sponsored by Swedish Orphan Biovitrum. Completed at 53 sites in 14 countries. Per ClinicalTrials.gov, last updated 2026-09-22.
Sponsored by Swedish Orphan Biovitrum · Observational
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.
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.
844 studies on the registry are indexed under Hemophilia A; 138 are open to participants now.
This study's enrollment of 427 is above the median of 81 across 310 observational studies indexed under Hemophilia A.
Browse Hemophilia A studies →Swedish Orphan Biovitrum is the lead sponsor of 82 studies on the registry; 6 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 15 (88%) have results posted.
Counted across the registry records on this site, refreshed daily.
Patients diagnosed with haemophilia A regardless of severity, of any gender and age, on factor treatment with Elocta according to usual clinical practice.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
All patients diagnosed with haemophilia A regardless of severity, on factor treatment with Elocta according to usual clinical practice.
Drug: ELOCTA
Extended half-life factor VIII product
Also known as: ELOCTATE, efmoroctocog alfa, rFVIIIFc
Joint health: Target joint development
Number of target joints
Time frame: 48 months
Joint health: Target joint resolution
Number of resolved target joints
Time frame: 48 months
Joint health: Target joint recurrence
Number of recurring target joints
Time frame: 48 months
Joint health: Annualised joint bleeding rate (AJBR) for treated bleeds
Number of joint bleeding events per year, for treated bleeds
Time frame: 48 months
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.
Time frame: 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.
Time frame: 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.
Time frame: 48 months
Effectiveness of Elocta: Annualised bleeding rate (ABR) for treated bleeds
Number of bleeding events per year, for treated bleeds
Time frame: 48 months
Effectiveness of Elocta: Annualised bleeding rate (ABR) for all bleeds
Number of bleeding events per year, for all bleeds
Time frame: 48 months
Effectiveness of Elocta: Occurrence of zero (0) joint bleeds
Percentage (%) of study population with zero (0) joint bleeds
Time frame: 48 months
Effectiveness of Elocta: Work productivity and impairment
Assessed by WPAI-SHP questionnaire
Time frame: 48 months
Effectiveness of Elocta: Quality of Life
Assessed by Haemo-QoL/Haem-A-QoL questionnaire
Time frame: 48 months
Effectiveness of Elocta: Physical activity level
Assessed by the Saltin-Grimby scale
Time frame: 48 months
Effectiveness of Elocta: Quality of Life
Assessed by EQ-5D-5L/EQ-5D-Y questionnaire
Time frame: 48 months
Effectiveness of Elocta: Severity of joint health
Assessed by Patient Global Impression of Severity of Joint Health questionnaire
Time frame: 48 months
Effectiveness of Elocta: FVIII plasma levels
Percentage (%) of FVIII plasma level
Time frame: 48 months
Effectiveness of Elocta: Usage of pain and anti-inflammatory medication
Assessed by medication dose
Time frame: 48 months
Effectiveness of Elocta: Usage of pain and anti-inflammatory medication
Assessed by total number of exposure days per medication
Time frame: 48 months
Usage of Elocta: Annualised injection frequency per subject
Assessed by prescription
Time frame: 48 months
Usage of Elocta: Annualised factor consumption per subject
Assessed by prescription \[IU/kg\]
Time frame: 48 months
Usage of Elocta: Adherence
Investigator assessed (Percentage (%))
Time frame: 48 months
Exploratory Objective
Influence of different Elocta prophylaxis regimens on long term joint health
Time frame: 48 months
Exploratory Objective: Impact of florio HAEMO on patients' sense of protection and their activity level
Results from questionnaire on impact of florio HAEMO tools on patients' sense of protection and their activity level
Time frame: 48 months
Exploratory Objective: Explore adherence to prescribed treatment regimen
florio HAEMO captured treatment adherence scores
Time frame: 48 months
Exploratory Objective: Explore relationship between predicted FVIII levels and physical activity
florio HAEMO captured predicted FVIII levels, florio HAEMO captured physical activity (type, duration, heart rate and steps)
Time frame: 48 months
Exploratory Objective: Explore timing of bleeds in relation to predicted FVIII levels and physical activity
florio HAEMO captured bleed data (timing, location, cause, treated/untreated), florio HAEMO captured injection data (time, dose), florio HAEMO captured predicted FVIII levels
Time frame: 48 months
Exploratory Objective: Explore the use of Florio to characterise pain and well-being
florio HAEMO captured pain data (time, location, cause, intensity), florio HAEMO captured well-being data
Time frame: 48 months
Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health
Target joint development, resolution and recurrence
Time frame: 48 months
Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health
Annualised joint bleeding rate (AJBR) for treated bleeds
Time frame: 48 months
Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health
Ultrasound (HEAD-US)
Time frame: 48 months
Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health
Clinical joint scoring (HJHS)
Time frame: 48 months
Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health
Annualised bleeding rate (ABR) (based on bleeding episodes assessed according to local practice) for all bleeds
Time frame: 48 months
Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health
Occurrence of zero (0) joint bleeds
Time frame: 48 months
Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health
Quality of Life assessed by Haemo-QoL/Haem-A-QoL
Time frame: 48 months
Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health
Physical activity level assessed by the Saltin-Grimby scale
Time frame: 48 months
Plan to share: No
No publications or documents are linked to this record.
Ran from Mar 2020 to primary completion in May 2026, 73 months.
Of 53 industry-sponsored observational studies under Hemophilia A, half reached primary completion within 58 months of starting (the median), counting those still running; a quarter within 34 and three quarters within 79. This study's 73 months is above that.
Counting every sponsor, half of 131 studies reached it within 45 months; a quarter within 25 and three quarters within 79.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.