Weekly shot may shield joints in hemophilia a patients
NCT ID NCT06752850
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether a once-weekly medication (efanesoctocog alfa) can improve or prevent joint damage in 37 people with moderate or severe hemophilia A. Participants receive the drug for 12 months, and doctors use ultrasound and MRI to check joint health. The goal is to see if this treatment reduces swelling and lowers the risk of joint bleeds.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 4
Runs after approval, following long-term safety and how well the treatment works in everyday use.
- Participants
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37 people
The number who actually took part.
- Started
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Dec 2024
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. Parents' or legally designated representatives' consent is required for patients who are \<18 years of age or unable to give consent, or as applicable per local laws. Patients who are \<18 years of age should provide assent in addition to the parents'/legally designated representatives' consent, if appropriate. 2. Male or female patients who are ≥12 years of age and diagnosed with moderate or severe haemophilia A (defined as ≤5% of normal FVIII clotting activity) at the time of signing the ICF. 3. A female patient is eligible to participate if she is not pregnant at enrolment and does not plan to become pregnant during the study. A woman of child-bearing potential (WOCBP) must have a negative highly sensitive serum pregnancy test at the Screening Visit. 4. Must have received prophylactic treatment per local label with any marketed FVIII product or emicizumab for ≥12 months prior to the Baseline Visit. 5. Have at least one eligible index joint (ankle, elbow, knee). 6. Have 12 months of documented pre-study treatment data on haemophilia prescriptions and on treated bleeding episodes prior to the Baseline Visit. 7. Willingness and the ability of the patient or their legally designated representative to complete training in the use of the study patient diary and to complete the diary throughout the study. Exclusion Criteria: 1. Blood clotting disorders other than haemophilia A 2. Already on efanesoctocog alfa treatment 3. Positive inhibitor result (assessed by local laboratory) from the Screening Visit, defined as ≥0.6 Bethesda units (BU)/mL. 4. History of inhibitors without successful immune tolerance induction (ITI) * Successful ITI is defined as: * Negative inhibitor titer (\<0.6 BU/mL) * FVIII recovery \> 66% of expected * FVIII half-life ≥ 6 hours 5. ITI performed within the last 2 years prior to the Baseline Visit. 6. Currently receiving treatment with any of the prohibited concomitant medications, as specified by the protocol. 7. Planned major orthopaedic procedure in any eligible index joint during the course of the study. 8. Patients are not eligible for participation in the study if they cannot undergo MRI assessments at the Baseline Visit. 9. Patients with known hypersensitivity to the active substance or to any of the excipients. 10. Patient not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures. 11. Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), within 3 months prior to inclusion in the study.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Sobi Investigational Site
Milan, Italy
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Sobi Investigational Site
Naples, Italy
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Sobi Investigational Site
Rozzano, Italy
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Sobi Investigational Site
Oslo, Norway
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Sobi Investigational Site
Barcelona, Spain
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Sobi Investigational Site
Madrid, Spain
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Sobi Investigational Site
Seville, Spain
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Sobi Investigational Site
Valencia, Spain
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Sobi Investigational Site
Gothenburg, Sweden
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Sobi Investigational Site
Malmö, Sweden
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Other studies related to the condition(s) this trial covers.
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