Hemophilia A, SeverePhase 3FREEDOMNCT05817812
A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa
A Phase 3 study testing Efanesoctocog alfa in people with Hemophilia A, Severe, run by Swedish Orphan Biovitrum at 30 sites in 14 countries.
Trial summary
FREEDOM is a multicentre, open-label, single-arm, phase 3b study in Europe that aims to enrol approximately 90 previously treated severe haemophilia A patients aged ≥12 years, currently on prophylaxis. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hemophilia A, Severe
- What is being tested?
- Efanesoctocog alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 93 participants
- Where
- 30 sitesin 14 countries
- Study dates
- 31 Jul 2023to 2 Jun 2026
- Phase
- Phase 3
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Previous treatment for haemophilia A with any marketed recombinant and/or plasma-derived FVIII for at least 150 EDs.
- Having received prophylactic treatment with any marketed recombinant and/or plasma FVIII or emicizumab per local label for at least 12 months preceding enrolment.
- Having 12 months documented pre-study treatment data regarding prophylactic treatment prescriptions and 6 months data on bleeding episodes prior to baseline visit.
- Willingness and ability to complete training in the use of the study ePD and to use the ePD in their own smartphone throughout the study.
- Willingness and ability to use the activity tracker provided by the sponsor to measure physical activity and heart rate.
- Be able and willing to administer efanesoctocog alfa intravenously at home.
Who cannot join
- Serious musculoskeletal and/or neurological impairment limiting the mobility and the physical ability to a degree that makes the patient unsuitable for the study as judged by the investigator.
- Other known coagulation disorder(s) in addition to haemophilia A.
- History and/or current positive inhibitor test defined as ≥0.6 BU/mL.
- Treatment with NSAIDs above the maximum dose specified in the prescribing information within 2 weeks prior to screening.
- Systematic treatment within 12 weeks prior to screening with chemotherapy and/or other immunosuppressive drugs.
- Treatment with an investigational product within 30 days or 5.5 half-lives prior to screening, whichever is longer.
- Major surgery within 12 weeks prior to screening or planned major orthopaedic surgery to occur during the study.
- At baseline visit, patients who have not been compliant in using the activity tracker.
Trial locations and contacts
30 sites in 14 countries.
30 sites in 14 countries
Italy5 sites
Investigational Site
Catanzaro
Investigational Site
Florence
Investigational Site
Naples
Investigational Site
Parma
Investigational Site
Rome
France4 sites
Investigational Site
Bordeaux
Investigational Site
Le Kremlin-Bicêtre
Investigational Site
Marseille
Investigational Site
Strasbourg
Germany4 sites
Investigational Site
Berlin
Investigational Site
Frankfurt
Investigational Site
Giessen
Investigational Site
Hamburg
Spain3 sites
Investigational Site
Barcelona
Investigational Site
Madrid
Investigational Site
Oviedo
United Kingdom3 sites
Investigational Site
Canterbury
Investigational Site
Newcastle upon Tyne
Investigational Site
Oxford
About this study
Official title: A Phase 3b Open-label Study Evaluating Physical Activity and Joint Health in Previously Treated Patients ≥12 Years With Severe Haemophilia A Treated With Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (Efanesoctocog Alfa) for 24 Months
FREEDOM is a multicentre, open-label, single-arm, phase 3b study in Europe that aims to enrol approximately 90 previously treated severe haemophilia A patients aged ≥12 years, currently on prophylaxis. After a run-in period of 30-45 days, patients will receive efanesoctocog alfa prophylaxis, 50 IU/kg once-weekly for 24 months (additional preventive dose not permitted). An activity tracker and an electronic patient diary will be used to collect data on physical activity, bleeds, factor dosing, pain, and injuries from screening throughout the study.
The primary objective is to describe changes in physical activities over 24 months on efanesoctocog alfa prophylaxis, with a primary endpoint of change from baseline in International Physical Activity Questionnaire (IPAQ) at month 24.
Secondary objectives include relationship between physical activity and other variables (bleeds, joint status, pain, injuries, and quality of life); changes in joint status as assessed by HEAD-US, HJHS and MRI; occurrence of bleeds, injuries, pain. Safety and tolerability of efanesoctocog alfa will also be evaluated.
- Conditions
- Hemophilia A, Severe
- Being tested
- Efanesoctocog alfa
- Keywords
- Blood coagulation disorder, Coagulation protein disorder, Haemophilia, FVIII, Efanesoctocog alfa
- Medical subject headings
- Hemophilia A, Blood Coagulation Disorders, Coagulation Protein Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Efanesoctocog alfaDrug
Also called BIVV001
Recombinant coagulation factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN)
| Study group | Role | What they receive |
|---|---|---|
| Efanesoctocog alfa | Experimental | All patients will be treated intravenously once weekly with 50 IU/kg efanesoctocog alfa Receives: Drug: Efanesoctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in International Physical Activity Questionnaire (IPAQ) scoring
The IPAQ is a validated, 7-day recall questionnaire measuring current levels of physical activity. Scoring of the IPAQ results in a continuous variable in the form of total MET-minutes per week. Higher scores means higher physical activity level.
Measured: Baseline and month 24
Secondary outcomes (42)
Change from baseline in International Physical Activity Questionnaire (IPAQ) scoring
The IPAQ is a validated, 7-day recall questionnaire measuring current levels of physical activity. Scoring of the IPAQ results in a continuous variable in the form of total MET-minutes per week. Higher scores means higher physical activity level.
Measured: Baseline and month 12
International Physical Activity Questionnaire (IPAQ) score
The IPAQ is a validated, 7-day recall questionnaire measuring current levels of physical activity. Scoring of the IPAQ results in a continuous variable in the form of total MET-minutes per week. Higher scores means higher physical activity level.
Measured: Baseline, month 6, 12, 18 and 24
Change in mean daily minutes in physical activity
Tracker-recorded
Measured: Run-in month, month 12 and 24
Mean daily minutes of physical activity
Tracker-recorded
Measured: Per 6-month period
Change in type of workouts
Patient and tracker reported
Measured: Run-in month, month 12 and 24
Change in frequency of workouts
Patient and tracker reported
Measured: Run-in month, month 12 and 24
Change in duration of workouts
Patient and tracker reported
Measured: Run-in month, month 12 and 24
Change in patient reported intensity of workouts
The patient will report workouts in a patient diary and rate the intensity on a 5 level scale. The levels are:
1 = easy, 2 = moderate, 3 = slightly challenging, 4 = exhausting, 5 = very exhausting.
The changes will be calculated on a intra-patient basis
Measured: Run-in month, month 12 and 24
Change in tracker recorded intensity of workouts
The patient will be provided with an activity tracker that will automatically record workouts when the patient is wearing the tracker. The intensity levels will be based on the heart rate and will be presented on a 4 level scale. The levels are:
1 = out of range, 2 = fat burn, 3 = cardio, 4 = peak
Measured: Run-in month, month 12 and 24
Mean value of patient and tracker reported type of workouts
Type of workout will be transcribed into a risk level of participating in a given activity for a haemophilia patient. Risk levels are based on a rating of 1-3 where level 1 represents a low risk of causing a bleeding episode and level 3 represents a high risk.
The mean value will be calculated per 6-month period
Measured: Per 6-month period
Mean value of frequency of workouts
Patient and tracker reported
Measured: Per 6-month period
Mean value of duration of workouts
Patient and tracker reported
Measured: Per 6-month period
Mean value of patient reported intensity of workouts
The patient will report workouts in a patient diary and rate the intensity on a 5 level scale. The levels are:
1 = easy, 2 = moderate, 3 = slightly challenging, 4 = exhausting, 5 = very exhausting.
The mean value will be calculated per 6-month period
Measured: Per 6-month period
Mean value of tracker recorded intensity of workouts
The patient will be provided with an activity tracker that will automatically record workouts when the patient is wearing the tracker. The intensity levels will be based on the heart rate and will be presented on a 4 level scale. The levels are:
1 = out of range, 2 = fat burn, 3 = cardio, 4 = peak
The mean value will be calculated per 6-month period
Measured: Per 6-month period
Change in mean daily number of steps
Tracker-recorded
Measured: Run-in month, month 12 and 24
Mean daily number of steps
Tracker-recorded
Measured: Per 6-month period
Achieving WHO-recommended levels of MVPA
Tracker-recorded
Measured: Run-in month, month 12 and 24
Occurrence of bleeds in relation to workouts
Measured: Baseline to month 24
Occurrence of pain in relation to workouts
Measured: Baseline to month 24
Occurrence of injuries in relation to workouts
Measured: Baseline to month 24
Occurrence of bleeding episodes impacting daily activity
Measured: Baseline to month 24
Change from baseline in Haemophilia Joint Health Score (HJHS) score
The six index joints (elbows, knees and ankles) will be examined and scored. The minimum score per joint is 0, the maximum score is 20. In addition, Gait is scored on a scale from 0 to 4 based on the number of skills that are not within the normal limits.
The total score will be the sum of scores across all six joints plus the gait score (range from 0 to 124, with 0 being normal and 124 being the most severe disease).
Measured: Baseline, month 6, 12, 18, 24
Change from baseline in Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US)
The HEAD-US joint score will be calculated for six joints, the right and left ankle, knee, and elbow joints. The specific joint score is made up of three item scores, disease activity (synovitis), disease damage of articular surfaces, and disease damage of subchondral bone. Specific joint score is the sum of the three item scores for each specific joint. Its values range from 0 (minimum) to 8 (maximum).The total score represents the sum of item scores for abnormalities detected.
Measured: Baseline, month 12 and 24
Change from baseline in haemophilic arthropathy assessed by the International Prophylaxis Study Group (IPSG) Magnetic Resonance Imaging (MRI) scale
The IPSG scale is made up of two subscores, one for soft-tissue changes and one for osteochondral changes. MRI joint score is the sum of the soft tissue and osteochondral subscores for each joint, ranging from 0-17 where 0 is absence of damage. MRI total score is the sum of joint scores across all joints.
Measured: Baseline, month 24
Target joint development (three or more spontaneous bleeds into a single joint within a consecutive 6-month period)
The number and proportion of patients with target joint development and the total number of target joint developed will be assessed.
Measured: Month 6, 12, 18, 24
Target joint resolution ( ≤2 bleeds into the joint within a consecutive 12-month period the joint is no longer considered a target joint)
The number and proportion of patients with target joint resolution and the total number of target joint resolutions will be assessed
Measured: Month 6, 12, 18, 24
Target joint recurrence (≥3 spontaneous bleeds in a single joint within any consecutive 6-month period after target joint resolution)
The number and proportion of patients with target joint recurrence and the total number of target joint recurrence will be assessed
Measured: Month 6, 12, 18, 24
Number of bleeding episodes
Measured: Per 6-month period and cumulative
Annualized bleeding rate (ABR)
Measured: Baseline to month 24
Total annualized efanesoctocog alfa consumption
Measured: Baseline to month 24
Number of injections and dose to treat a bleeding episode
Measured: Baseline to month 24
Pain reported via electronic patient diary
The patient will report pain (cause of pain, location and intensity. The intensity will reported as mild, moderately low, moderately high, severe and worst possible.
Measured: Baseline to month 24
Pain intensity reported in the Patient-Reported Outcomes Measurement Information System (PROMIS) Scale - Pain Intensity 3a
The PROMIS Pain Intensity instrument assesses pain intensity on a 5-point Likert scale, ranging from 1 (had no pain) to 5 (very severe)
Measured: Baseline, month 6, 12, 18, 24
Pain interference reported in the Patient-Reported Outcomes Measurement Information System (PROMIS) scale - Pain interference 6a
The PROMIS Pain Interference 6a is a patient-assessed instrument and is to measure pain interference on a 5-point Likert scale, ranging from 1 (not at all) to 5 (very much)
Measured: Baseline, month 6, 12, 18, 24
Quality of life assessed by EuroQoL 5-dimension 5-level (EQ-5D-5L)
EQ-5D-5L assess 5 dimensions of health outcome (mobility, self-care, usual activities, pain/discomfort, anxiety/depression). Each of the dimensions in the EQ-5D-5L questionnaire is divided into 5 levels:
- Level 1: indicating no problem
- Level 2: indicating slight problems
- Level 3: indicating moderate problems
- Level 4: indicating severe problems
- Level 5: indicating unable to/extreme problems Patients will select a level for each dimension that best describes their health status for that dimension. A 5 digit code will be obtained, made up of the response for each dimension. This code will then be converted to an index score.The EQ-5D-5L VAS is an overall assessment of health on a scale from 1-100 that the patient provides at the end of the EQ-5D-5L questionnaire. 100 represents the best health you can imagine and 0 represents the worst health you can imagine.
Measured: Baseline, month 6, 12, 18, 24
Assessment of treatment preference
This will be assessed with the Treatment Preference Survey that consists of 2 questions on perceived impact of treatment.
Measured: 24 months
The occurrence of adverse events and serious adverse events
Measured: From screening to month 24
The occurrence of clinically significant changes
Assessed by physical examination, vital signs and laboratory tests
Measured: Baseline, month 6, 12, 18, 24
Development of inhibitors (neutralizing antibodies directed against FVIII)
Determined via the Nijmegen modified Bethesda assay
Measured: Baseline, month 6, 12, 18, 24
The occurrence of thrombotic and embolic events
Measured: From screening to month 24
Number of non-study medical care encounters
The number and proportion of patients having non-study medical care encounter visits will be described, as well as the corresponding total number of visits.
Measured: From baseline to month 24
Duration of non-study medical care encounters
The duration of non-study medical care encounter visits will be described.
Measured: From baseline to month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2023
- Study start31 Jul 2023
- Primary completion2 Jun 2026
- Study completion2 Jun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A, SevereStudies 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 93 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Syneos Health
- Responsible party
- Sponsor
- Investigators
- Study Physician, MD, PhD, Study director, Swedish Orphan Biovitrum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05817812
- Sponsor's protocol ID
- Sobi.BIVV001-001
- Acronym
- FREEDOM
- Status last verified
- Jul 2026
- First submitted
- 21 Mar 2023
- Last updated
- 2 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sobi's data sharing criteria and process for requesting access can be found at: https://www.sobi.com/en/policies
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3b Open-label Study Evaluating Physical Activity and Joint Health in Previously Treated Patients ≥12 Years With Severe Haemophilia A Treated With Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (Efanesoctocog Alfa) for 24 Months
- ClinicalTrials.gov ID
- NCT05817812
- Acronym / sponsor ID
- FREEDOM / Sobi.BIVV001-001
- Conditions
- Hemophilia A, Severe
- Keywords
- Blood coagulation disorder, Coagulation protein disorder, Haemophilia, FVIII, Efanesoctocog alfa
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 93 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Collaborators
- Syneos Health
- Study start
- 31 Jul 2023 (actual)
- Primary completion
- 2 Jun 2026 (actual)
- Study completion
- 2 Jun 2026 (actual)
- First posted
- 18 Apr 2023 (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.
FREEDOM is a multicentre, open-label, single-arm, phase 3b study in Europe that aims to enrol approximately 90 previously treated severe haemophilia A patients aged ≥12 years, currently on prophylaxis. After a run-in period of 30-45 days, patients will receive efanesoctocog alfa prophylaxis, 50 IU/kg once-weekly for 24 months (additional preventive dose not permitted). An activity tracker and an electronic patient diary will be used to collect data on physical activity, bleeds, factor dosing, pain, and injuries from screening throughout the study.
The primary objective is to describe changes in physical activities over 24 months on efanesoctocog alfa prophylaxis, with a primary endpoint of change from baseline in International Physical Activity Questionnaire (IPAQ) at month 24.
Secondary objectives include relationship between physical activity and other variables (bleeds, joint status, pain, injuries, and quality of life); changes in joint status as assessed by HEAD-US, HJHS and MRI; occurrence of bleeds, injuries, pain. Safety and tolerability of efanesoctocog alfa will also be evaluated.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Efanesoctocog alfa | Experimental | Drug: Efanesoctocog alfaAll patients will be treated intravenously once weekly with 50 IU/kg efanesoctocog alfa |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Previous treatment for haemophilia A with any marketed recombinant and/or plasma-derived FVIII for at least 150 EDs.
- Having received prophylactic treatment with any marketed recombinant and/or plasma FVIII or emicizumab per local label for at least 12 months preceding enrolment.
- Having 12 months documented pre-study treatment data regarding prophylactic treatment prescriptions and 6 months data on bleeding episodes prior to baseline visit.
- Willingness and ability to complete training in the use of the study ePD and to use the ePD in their own smartphone throughout the study.
- Willingness and ability to use the activity tracker provided by the sponsor to measure physical activity and heart rate.
Key exclusion criteria (8 listed)
- Serious musculoskeletal and/or neurological impairment limiting the mobility and the physical ability to a degree that makes the patient unsuitable for the study as judged by the investigator.
- Other known coagulation disorder(s) in addition to haemophilia A.
- History and/or current positive inhibitor test defined as ≥0.6 BU/mL.
- Treatment with NSAIDs above the maximum dose specified in the prescribing information within 2 weeks prior to screening.
- Systematic treatment within 12 weeks prior to screening with chemotherapy and/or other immunosuppressive drugs.
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 |
|---|---|---|
| Efanesoctocog alfaAlso: BIVV001Recombinant coagulation factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN) | Drug | Efanesoctocog alfa |
Primary outcome
Change from baseline in International Physical Activity Questionnaire (IPAQ) scoring
Time frame: Baseline and month 24
The IPAQ is a validated, 7-day recall questionnaire measuring current levels of physical activity. Scoring of the IPAQ results in a continuous variable in the form of total MET-minutes per week. Higher scores means higher physical activity level.
42 secondary outcomes
Change from baseline in International Physical Activity Questionnaire (IPAQ) scoring
Time frame: Baseline and month 12
International Physical Activity Questionnaire (IPAQ) score
Time frame: Baseline, month 6, 12, 18 and 24
Change in mean daily minutes in physical activity
Time frame: Run-in month, month 12 and 24
Mean daily minutes of physical activity
Time frame: Per 6-month period
Change in type of workouts
Time frame: Run-in month, month 12 and 24
Change in frequency of workouts
Time frame: Run-in month, month 12 and 24
Change in duration of workouts
Time frame: Run-in month, month 12 and 24
Change in patient reported intensity of workouts
Time frame: Run-in month, month 12 and 24
Change in tracker recorded intensity of workouts
Time frame: Run-in month, month 12 and 24
Mean value of patient and tracker reported type of workouts
Time frame: Per 6-month period
Mean value of frequency of workouts
Time frame: Per 6-month period
Mean value of duration of workouts
Time frame: Per 6-month period
Mean value of patient reported intensity of workouts
Time frame: Per 6-month period
Mean value of tracker recorded intensity of workouts
Time frame: Per 6-month period
Change in mean daily number of steps
Time frame: Run-in month, month 12 and 24
Mean daily number of steps
Time frame: Per 6-month period
Achieving WHO-recommended levels of MVPA
Time frame: Run-in month, month 12 and 24
Occurrence of bleeds in relation to workouts
Time frame: Baseline to month 24
Occurrence of pain in relation to workouts
Time frame: Baseline to month 24
Occurrence of injuries in relation to workouts
Time frame: Baseline to month 24
Occurrence of bleeding episodes impacting daily activity
Time frame: Baseline to month 24
Change from baseline in Haemophilia Joint Health Score (HJHS) score
Time frame: Baseline, month 6, 12, 18, 24
Change from baseline in Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US)
Time frame: Baseline, month 12 and 24
Change from baseline in haemophilic arthropathy assessed by the International Prophylaxis Study Group (IPSG) Magnetic Resonance Imaging (MRI) scale
Time frame: Baseline, month 24
Target joint development (three or more spontaneous bleeds into a single joint within a consecutive 6-month period)
Time frame: Month 6, 12, 18, 24
Target joint resolution ( ≤2 bleeds into the joint within a consecutive 12-month period the joint is no longer considered a target joint)
Time frame: Month 6, 12, 18, 24
Target joint recurrence (≥3 spontaneous bleeds in a single joint within any consecutive 6-month period after target joint resolution)
Time frame: Month 6, 12, 18, 24
Number of bleeding episodes
Time frame: Per 6-month period and cumulative
Annualized bleeding rate (ABR)
Time frame: Baseline to month 24
Total annualized efanesoctocog alfa consumption
Time frame: Baseline to month 24
Number of injections and dose to treat a bleeding episode
Time frame: Baseline to month 24
Pain reported via electronic patient diary
Time frame: Baseline to month 24
Pain intensity reported in the Patient-Reported Outcomes Measurement Information System (PROMIS) Scale - Pain Intensity 3a
Time frame: Baseline, month 6, 12, 18, 24
Pain interference reported in the Patient-Reported Outcomes Measurement Information System (PROMIS) scale - Pain interference 6a
Time frame: Baseline, month 6, 12, 18, 24
Quality of life assessed by EuroQoL 5-dimension 5-level (EQ-5D-5L)
Time frame: Baseline, month 6, 12, 18, 24
Assessment of treatment preference
Time frame: 24 months
The occurrence of adverse events and serious adverse events
Time frame: From screening to month 24
The occurrence of clinically significant changes
Time frame: Baseline, month 6, 12, 18, 24
Development of inhibitors (neutralizing antibodies directed against FVIII)
Time frame: Baseline, month 6, 12, 18, 24
The occurrence of thrombotic and embolic events
Time frame: From screening to month 24
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 Jul 2026.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sobi's data sharing criteria and process for requesting access can be found at: https://www.sobi.com/en/policies
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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, PhD study director
Swedish Orphan Biovitrum
30 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Efanesoctocog alfa in people with Hemophilia A, Severe, run by Swedish Orphan Biovitrum at 30 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 6 inclusion and 8 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 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in Italy, France, Germany, Spain, United Kingdom 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Change from baseline in International Physical Activity Questionnaire (IPAQ) scoring (time frame: Baseline and month 24). 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 Jul 2026.