At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Efanesoctocog alfa (drug)
- How long the study runs
- Study runs about 43 months (dates as stated)
- About the drug or intervention
- Efanesoctocog alfa — drug: Participant in this study must be prescribed efanesoctocog alfa prophylactic treatment per their standard of care from their physician.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- Not specified
- Who
- All
- Number of participants
- 252
- Started
- 2025-05-21
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for hemophilia a
- • Phase4 - 252 participants
- • The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting
Who can take part?
- • Adults
- • Diagnosed with hemophilia a
Where?
- • Birmingham - Sobi Investigational Site
- • Cardiff - Sobi Investigational Site
- • London - Sobi Investigational Site
- • Manchester - Sobi Investigational Site
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
More detail
This is a retrospective-prospective, low-interventional study to evaluate the long-term effectiveness of real-world prophylaxis with efanesoctocog alfa on joint health in people with haemophilia A. Participants in the study must be prescribed efanesoctocog alfa at the discretion of their physicians within 6 months prior to, or at the time of, enrolment. The decision to initiate treatment with efanesoctocog alfa shall be made independently from the decision to include the patients in the study. A broad haemophilia A population (e.g. all severities \[mild to severe\], all ages, patients with comorbidities and/or concomitant use of anticoagulant/antiplatelet drugs, patients with previous inhibitors to Factor FVIII \[FVIII\]) will be eligible to enrol. The study will require mandatory on-site visits with joint assessments. A retrospective data collection on patients' haemophilia, medical, and surgical history will be performed, including previous haemophilia treatment and treated bleeding episodes from 12 months prior to initiating treatment with efanesoctocog alfa until enrolment. The study will start with the enrolment visit during a patient's routine visit to the hospital, to check whether the patient fulfils all the inclusion criteria and none of the exclusion criteria. Study participants will have yearly on-site visits for 24 months or 36 months, depending on the time of enrolment in relation to study closure. Patients will be followed in the study as long as they are treated with efanesoctocog alfa prophylaxis which would be at the physicians' discretion. Patients will also need to agree to document any bleeding episodes they may have during the study in a study-specific paper diary. Joint assessment, Haemophilia Joint Health Score (HJHS), and completion of Patient-reported outcomes (PROs) will be conducted during the on-site visits. For patients included in the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) group, ultrasound joint assessment will also be done at the yearly visits. The end of study (EoS) visit can be performed at the 24-month visit or the 36-month visit, depending on the time of enrolment. The target is to enroll approximately 250 participants.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: Not specified
- Who can join: All genders
Biomarkers mentioned
Treatment history
Treatments you must have had:
- ✓ received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months
What the study is looking for
- ✓Male or female patient with a diagnosis of haemophilia A.
- ✓Capable of giving signed agreement to take part, which includes compliance with the requirements and restrictions listed...
- ✓Prescribed with efanesoctocog alfa prophylactic treatment within 6 months prior to study enrolment or at the latest...
- ✓Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being...
- ✓Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs).
Who cannot take part
- ✗Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A.
- ✗Any positive FVIII inhibitor result (defined as inhibitor titre ≥0.6 Bethesda unit \[BU\]/mL) from the medical...
- ✗Patient not suitable for participation, whatever the reason, as judged by the Investigator, e.g., patient is not...
See the full criteria
Where Is This Study? (4 UK sites)
Sobi Investigational Site
Birmingham, United Kingdom
Sobi Investigational Site
Cardiff, United Kingdom
Sobi Investigational Site
London, United Kingdom
Sobi Investigational Site
Manchester, United Kingdom
